FDA Approved Drugs in 2018

January

Lutetium Lu 177 dotatate

Lutethera (Lutetium Lu 177 dotatate) injection for intravenous use has been approved by US FDA for treating adult patients with somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors including the midgut, foregut and hindgut neuroendocrine tumors.

The safety and efficacy profile of Lutetium Lu 177 dotatate was carried out in two studies such as NETTER-1 and ERASMUS. The study NETTER-1 which was a randomized, multicenter, open-label and active-controlled trial involved 111 patients with advanced or progressive midgut neuroendocrine tumors with an additional 22 patients in a non-randomized pharmacokinetic study model. ERASMUS included 1214 patients in an single-arm, international, open-label trial.

Three complete responses were observed in the clinical studies and the median duration of response in the 58 responding patients was 35 months.

February

Apalutamide

US FDA approved Erleada (Apalutamide) tablets for treating non-metastatic castration-resistant prostate cancer (NM-CRPC). Apalutamide, an androgen receptor inhibitor is the first drug approved for the treatment of NM-CRPC.

The effectiveness of Apalutamide was evaluated in a multicenter, double-blind, randomized and placebo controlled clinical trial called SPARTAN. This trial involved 1207 patients in which patients received apalutamide 240 mg orally or placebo once a day. The main efficacy outcome of the study was the metastasis-free survival and significant improvement in metastasis-free survival was showed by the patients who were given apalutamide when compared to placebo.

The additional efficacy endpoints were progression-free survival (PFS), time to metastasis (TTM), time to symptomatic progression, and overall survival (OS).

Bictegravir, Emtricitabine and tenofovir alafenamide

Biktarvy (Bictegravir, Emtricitabine and tenofovir alafenamide) tablets for oral use was approved by US FDA for treating HIV-1 infection in adult patients who have not received any antiretroviral therapy or in virologically suppressed adults.

Four clinical trials were carried out to determine the safety and efficacy profile of the three drug combination of bictegravir, Emtricitabine and tenofovir alafenamide. Trial 1489 and Trial 1490 were carried out in adults with no antiretroviral therapy. Trial 1844 and Trial 1878 were carried out in population of virologically suppressed adults. Both the trials were randomized and active controlled trial.

The major adverse reactions associated with Bictegravir, Emtricitabine and tenofovir alafenamide were found to be of grade 1. Acute exacerbation of hepatitis B may occur after the treatment with Biktarvy and patients should be strictly monitored and treated with supportive care.

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Tezacaftor and Ivacaftor

Symdeko (Tezacaftor, Ivacaftor) tablets for oral use was approved by US FDA for treating cystic fibrosis in patients 12 years of age and above who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator gene. The effectiveness of tezacaftor and ivacaftor in patients with cystic fibrosis were evaluated in three Phase 3 double-blind and placebo-controlled trials.

Trial 1 evaluated 504 patients, trial 2 involved 244 patients and trial 3 studied 168 patients. Patients in all trials were continued on their standard-of-care cystic fibrosis therapies involving bronchodilators, hypertonic saline, inhaled antibiotics, and dornase alfa. The pharmacokinetic parameters of tezacaftor and ivacaftor were similar between the healthy volunteers and the patients with cystic fibrosis.

March

Ibalizumab-uiyk

Trogarzo (Ibalizumab-uiyk) injection for intravenous use was approved by US FDA to use in combination with other antiretroviral drugs for treating human immunodeficiency virus type 1 infection in adults who showed multidrug-resistant to the current antiretroviral regimen.

Trial TMB-301 was conducted in 40 patients who were heavily treatment-experienced HIV-infected subjects. The trial TMB-301 was a single arm and multicenter clinical trial which was composed of three discrete periods. These periods were control period from day 0 to day 6, functional monotherapy period from day 7 to day 13, and the maintenance period from day 14 to week 25.

Trogarzo as a maintenance dose of 800 mg was administered every two weeks from day 21 to week 25.Trogarzo in combination with other antiretroviral agents showed significant reduction in viral load and increased CD4 count.

Tildrakizumab-asmn

Ilumya (Tildrakizumab-asmn) injection was approved by US FDA for treating adults with moderate to severe plaque psoriasis who are candidates for systemic therapy or phototherapy. The dose was administered as a 100 mg subcutaneous injection for every 12 weeks during the study.

The efficacy of tildrakizumab-asmn was studied in two clinical trials reSURFACE 1 and reSURFACE 2 which involved 926 patients in which 616 adults were treated with tildrakizumab-asmn and 310 with placebo.

Patients treated with tildrakizumab-asmn showed significant clinical improvement with psoriasis reduction by 75% and Physician Global Assessment score of either clear or minimal when compared with placebo.

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April

Burosumab-twza

Crysvita (Burosumab-twza) is a fibroblast growth factor 23 blocking antibody injection was approved by FDA for subcutaneous use in treating X-linked hypophosphatemia (XLH) in adult and pediatric patients who were 1 year of age and older.

The minimum dose is 10 mg and the maximum dose can be given upto 90 mg which can be administered for every two weeks in pediatric patients from 1 year to less than 18 years and for every four weeks in adults.

Burosumab-twza was evaluated in 65 pediatric patients with X-linked hypophosphatemia in two clinical studies. Study 1 included 52 prepubescent patients and study 2 involved 13 pediatric patients. Study 3 is a randomized, placebo-controlled, double blind study involved 134 adult patients with XLH and study 4 is a 48 week single arm study which included 14 adult patients. During these studies the serum phosphorous levels were assessed regularly and healing of bone fractures were also observed.

Fosnetupitant and palonosetron

Akynzeo (fosnetupitant and palonosetron) injection for intravenous use was approved by FDA to use in combination with dexamethasone for the prevention of acute and delayed nausea and vomiting in adults associated with initial and repeat courses involving highly emetogenic cancer chemotherapy.

The efficay and safety of fosnetupitant and palonosetron were studied in five clinical studies. Patients must be advised to consult the physician if any allergic reactions or serotonin syndrome occurs. Women of childbearing age should not take fosnetupitant and palonosetron as it can cause harm to the fetus.

Safety and efficacy were not studied in pediatric patients and should be used in caution in the elderly because of the greater frequency of reduced hepatic, kidney or cardiac condition.

Fostamatinib

Tavalisse (Fostamatinib) tablets for oral use was approved by FDA for treating thrombocytopenia in adults with chronic immune thrombocytopenia who showed a treatment failure with previous treatments.

The efficacy and safety of fostamatinib was studied in two placebo-controlled studies (FIT-1 and FIT-2) and in a open-label extension study (FIT-3). FIT-1 study involved 76 patients, FIT-2 involved 74 patients and FIT-3 involved 123 patients. All patients were studied for stable response and 18 patients maintained the platelet count of at least 50 × 109 /L for 12 months or even longer.

Patients taking fostamatinib should be advised to monitor blood pressure, liver enzymes level, and complete blood count during the treatment and adequate supportive measures are needed if the symptoms becomes severe.

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May

Avatrombopag

Doptelet (Avatrombopag) tablets for oral use was approved by FDA on May 2018 for treating adult patients with thrombocytopenia associated with a severe liver disease who are scheduled to undergo a medical or dental procedure.

The safety profile of avatrombopag were studied in two (ADAPT-1 and ADAPT-2) placebo-controlled, double-blind, international, identically designed, and international studies.

Nearly 430 patients were involved in these studies and they were presented with chronic liver disease and thrombocytopenia. Among these, 274 patients were treated with avatrombopag and 156 patients were treated with placebo daily for 5 consecutive days before a scheduled procedure. The most common but severe side-effect of avatrombopag is abnormally low level of sodium in the blood which should be managed appropriately.

Baricitinib

Olumiant (Baricitinib) tablets for oral use were approved by FDA on May 31, 2018, for treating adult patients suffering from moderate to severely active rheumatoid arthritis who showed a loss of response with one to more tumor necrosis factor antagonist therapies. Baricitinib can also be prescribed alone or in combination with methotrexate. The recommended dose of baricitinib is 2 mg once daily.

The safety and effectiveness of baricitinib were evaluated in two confirmatory phase 3 trials which were double-blind, randomized and multicenter studies. Patients showed greater improvement from baseline in physical functioning along with other health-related outcomes when compared to placebo. The physical function response was measured by Health Assessment Questionnaire-Disability Index (HAQ-DI).

Erenumab-aooe

Aimovig (Erenumab-aooe) is a human monoclonal antibody approved by FDA on May 2018 to use as a subcutaneous injection for the preventive treatment of migraine in adults. The effectiveness of erenumab-aooe were evaluated in two studies involving patient groups with episodic migraine and the other study involved patients with chronic migraine.

All the three studies did not involve patients with medications overuse headache and patients with myocardial infarction. Erenumab-aooe showed statistically significant improvements when compared with placebo.

Erenumab-aooe should not be used in children and patients more than 65 years of age as the safety and effectiveness were not studied in those groups.

Lofexidine

Lucemyra (Lofexidine) tablets for oral use were approved by FDA on May 2018, for minimising the severity of opioid withdrawal symptoms. It was earlier approved in the UK in 1992. It also helps in treating patients where an abrupt opioid discontinuation is required.

Two double-blind, randomized, and placebo-controlled clinical trials were conducted to evaluate the efficacy of lofexidine. The efficacy, safety, and the dose-response were studied in two different studies such as study-NCT01863186 (229 patients on 2.16 mg and 222 were on 2.88 mg) and study-NCT00235729 (264 patients on 2.88 mg) conducted among inpatients in the United States.

Both the groups met the DSM-IV criteria for opioid dependence who were physically dependent on hydrocodone, oxycodone, and heroin and showed significant results of SOWS-Gossop scores when compared with placebo.

Pegvaliase-pqpz

Palynziq (pegvaliase-pqpz) injection for subcutaneous use was approved by FDA on May 2018 for treating phenylketonuria (PKU), a rare genetic disease with a high concentration of phenylalanine in adults. Two studies (study 301 and 302) were conducted to evaluate the safety, tolerability, and efficacy of pegvaliase-pqpz.

Patients should be examined for the blood concentration of phenylalanine during the treatment with pegvaliase-pqpz. Animal data on pegvaliase-pqpz therapy showed some toxicity studies on foetus. Clinical trials showed 83% of patients experienced episodes of joint pain, back pain, pain in the extremities, and neck pain. Patients also reported injection site reactions and generalized skin reactions.

Patients should be treated with antiallergic agents, corticosteroids and fever medications to overcome the hypersensitivity reactions during treatment with pegvaliase-pqpz.

Sodium zirconium cyclosilicate

Lokelma (Sodium zirconium cyclosilicate) was approved by FDA on May 18, 2018, to use as an oral suspension for treating adult patients with hyperkalemia or high potassium level in the blood. It act as a selective potassium binder in the gastrointestinal tract prescribed for treating adult patients suffering from hyperkalemia or high potassium level in the blood.

Sodium zirconium cyclosilicate should not be recommended as an emergency treatment for life-threatening hyperkalemia. It is generally recommended to take drugs either two hours before or two hours after the dose of sodium zirconium cyclosilicate to avoid the loss of effectiveness of those medications.

June

Binimetinib

Mektovi (Binimetinib) tablets was approved by FDA on June 27, 2018, to use in combination with encorafenib for treating adult patients with a specific type of skin cancer known as melanoma which cannot either be removed by surgery or the cancer that has moved to other parts of the body. Binimetinib is used to treat melanoma with a BRAF V600E or V600K gene mutation as confirmed by an FDA approved test.

Binimetinib should not be used for children as safety and effectiveness of binimetinib were not studied in patients of these age groups. Women of childbearing age should perform a pregnancy test and must take binimetinib only when the pregnancy test shows negative.

Cannabidiol

Epidiolex (Cannabidiol) solution for oral use was approved by FDA on June 25, 2018, prescribed for treating seizures in children above two years and older who are associated with Lennox-Gastaut syndrome (stiffening of muscles or loss of muscle tension) or Dravet syndrome (seizures due to high fever). Cannabidiol is marijuana plant-derived highly purified drug should be dispensed against a valid prescription from a registered medical practitioner.

Encorafenib

Braftovi (Encorafenib) capsules for oral use is prescribed with binimetinib for treating advanced melanoma which cannot either be removed by surgery or cancer that has moved to other parts of the body in adult patients with BRAF V600E or V600K mutation confirmed by an FDA-approved test. Tumor specimens should be tested for the presence of BRAF V600E or V600K mutation before starting the treatment with encorafenib. A pregnancy test should be done on women of childbearing age before the therapy and the treatment with encorafenib should be started only if the pregnancy test reads negative. Men of reproductive potential should plan to store their sperms in a sperm bank before starting encorafenib treatment as it may affect their fertility status.

Moxidectin

Moxidectin tablets for oral use is prescribed to treat onchocerciasis or river blindness in patients aged 12 years and above caused by the parasite Onchocerca volvulus. Onchocerciasis is the second most common cause of blindness occurs due to an infection. It is an anthelmintic or antihelminthic agent works by attacking the parasite by binding to specific ion channel resulting in paralysis of the parasite without affecting the host. However, moxidectin will not kill the parasite.

River blindness or onchocerciasis is an eye and skin disease transmitted by repeated bites of Simulium type infected blackflies thereby causing severe itching, disfiguring conditions of the skin, and loss of vision. The recommended dose of moxidectin is 8 mg of moxidectin (taken as four 2mg tablets onetime) as a single dose.

Plazomicin

Zemdri (Plazomicin) was approved by FDA on June 25, 2018, for treating adult patients with complicated urinary tract infections (cUTI) including pyelonephritis caused by specific microorganisms such as Escherichia coli, Klebsiella pneumoniae, Proteus mirabilis, and Enterobacter cloacae. Plazomicin may cause neuromuscular blockade mainly in patients with a history of neuromuscular disorders such as myasthenia gravis and can increase the risk of kidney damage,and hearing loss. Plazomicin should not be given to pregnant women as it may harm the fetus.

July

Elagolix

Orilissa (Elagolix) tablets for oral use are used for the management of moderate to severe pain associated with endometriosis, a painful condition in which the endometrial tissue grows outside the uterus. Elagolix helps to reduce the symptoms of menstrual and non-menstrual pelvic pain and pain that occurs during sexual intercourse.

Perform a pregnancy test before starting elagolix tablets and initiate the treatment only if the test is negative to avoid the risk of early pregnancy loss. Taking supplements containing calcium and vitamin D during elagolix treatment may be very helpful in strengthening the bones.

Fish oil triglycerides

Omegaven (Fish oil triglycerides) is used as a nutritional supplement containing omega 3 fatty acids given into the vein directly in patients who cannot take food by mouth. It serves as total parenteral nutrition which delivers the essential nutrients directly into the bloodstream. It is a mixture of highly refined fish oil containing omega-3 fatty acids such as eicosapentaenoic acid (EPA) and docosahexaenoic acid (DHA) with alpha-tocopherol, a form of vitamin E known for its antioxidant property.

Fish oil triglycerides are used to provide parenteral nutrition in patients who suffer from Parenteral Nutrition Associated Cholestasis (PNAC). Fish oil triglycerides should not be given to premature infants, newborns, infants, children below 11 years, and patients with liver or kidney dysfunction as the safety and effectiveness were not studied in these groups.

Ivosidenib

Tibsovo (Ivosidenib) tablets are used to treat acute myeloid leukemia (AML), a cancer of the blood and bone marrow in adult patients with a specific IDH1 gene alteration whose condition worsens after treatment or resistant to other medicines. The mutation or a permanent gene alteration in IDH1 that causes AML is confirmed by performing Real Time IDH1 Assay, an FDA-approved test to detect IDH1 mutations.

The dose of ivosidenib can be temporarily stopped or reduced to 250 mg once daily, if abnormal heart rhythm, nerve damage, and symptoms of differentiation syndrome along with an increase in the number of white blood cells occur.Treatment with the usual adult dose can be started once the side effects are mild or reduced greatly. Also the treatment with ivosidenib can be permanently stopped if the symptoms and signs are life-threatening.

Lusutrombopag

Mulpleta (Lusutrombopag) tablets are used to treat abnormally low platelet count (thrombocytopenia) in patients with a severe liver disease who are scheduled to undergo a surgical procedure. Low platelet counts can result in poor clotting which leads to uncontrollable bleeding during surgery. This may delay the procedure or even cancel the treatment because of the risk to the patient.

Lusutrombopag tablets taken once daily for a week may improve the platelet levels in patients with liver disease and therefore reduce the need for platelet transfusions before the surgery. Lusutrombopag should not be given to pregnant and breastfeeding women as it may harm the baby.

Tafenoquine

Krintafel (Tafenoquine) is an antimalarial drug used to prevent the disease the from returning in patients affected by malaria caused by Plasmodium vivax. It was approved by FDA in July 2018 to use in patients of age 16 years and above who receive treatments for P.vivax infection.

Tafenoquine should never be used to treat Plasmodium vivax but can be given together with other drugs to treat Plasmodium vivax infection. Patients suffering from a deficiency of Glucose-6-phosphate dehydrogenase (G6PD) should not take tafenoquine due to the risk of breakdown of red blood cells. The recommended dose of tafenoquine is 300 mg which is taken as two tablets of tafenoquine 150 mg as a single dose.

Tecovirimat

TPOXX (Tecovirimat) can be used to treat human smallpox disease in adult and pediatric patients who weigh at least 13 kg by acting particularly on orthopoxvirus VP37 envelope wrapping protein. Tecovirimat has been given a fast track status and approved by FDA as it has a high level of specificity and selectivity against orthopoxvirus, a specific virus responsible for causing the disease.

The dose of tecovirimat is calculated based on the body weight of the patients. Tecovirimat capsule should be taken immediately or within 30 minutes after eating a full meal with a moderate to high fat. Elderly patients should be carefully observed during the treatment with tecovirimat as the occurrence of side effects are estimated to be high which can affect patients quality of life.

August

Cenegermin-bkbj

Oxervate (Cenegermin-bkbj) eye drops are prescribed to treat neurotrophic keratitis, a rare degenerative disease that affects the cornea by causing damage to the trigeminal nerve that supplies the surface of the eye resulting in loss of sensation and poor healing. The loss of corneal sensation may affect corneal health leading to corneal thinning, ulceration, and perforation in severe cases. It should be used only in adults with moderate to severe disease. Cenegermin repairs the damage caused to the eye surface and helps to restore the normal healing processes in the eye.

The recommended dose of cenegermin is one drop to be instilled into the affected eye every two hours, six times a day continued for eight weeks. An eye specialist should supervise the treatment with cenegermin. The most common side effects are swelling and pain in the eye, watery eyes or increased lacrimation, eyelid pain, and sensation of a foreign body in the eye. Neurotrophic keratitis is believed to affect less than five people in 10,000 individuals; therefore FDA has granted orphan drug designation to cenegermin.

Doravirine

Pifeltro (Doravirine) tablets are prescribed along with other antiretroviral medicines to treat human immunodeficiency virus- 1 (HIV-1) infection in adult patients who are not treated before with HIV-1 treating medications. HIV-1 is the virus that is responsible for causing AIDS (Acquired Immune Deficiency Syndrome). Doravirine should not be given to pregnant and breastfeeding women as it can harm the baby.

The recommended dose of doravirine is 100 mg to be taken once a day either with or without food. If doravirine is co-administered with rifabutin, doravirine tablets should be taken twice daily. The most common side effects of doravirine are dizziness, nausea, headache, tiredness, loose stools, stomach pain, rashes, and abnormal dreams.

Lanadelumab-flyo

Takhzyro (Lanadelumab-flyo) is a monoclonal antibody used to treat type I and II hereditary angioedema (HAE), a rare and severe genetic disease that results in repeated attacks of swelling of the face, arms, airway, intestinal tract, and the legs. Type I is the most common type which accounts for about 85 percent of the cases. Lanadelumab is used in patients 12 years and above with HAE who have either low levels or poor function of a specific protein (C1-INH) in the body. The most common side effects that can occur during lanadelumab treatment are headache, injection site reactions, dizziness, loose stools, upper respiratory functions, muscle pain, and rashes.

HAE can be first noticed in childhood and affects severely after puberty. HAE affects both men and women equally as some patients get frequent attacks in a month while some patients do not show any attacks for months. It is estimated to affect approximately one in 50,000 people. FDA granted orphan designation to lanadelumab.

Migalastat

Galafold (Migalastat) capsules are prescribed to treat Fabry disease in adult patients whose genetic mutation is confirmed by a specific lab test. Fabry disease is a rare and an inherited disorder but a severe genetic illness. This disease occurs due to the accumulation of a particular type of fat called globotriaosylceramide (GL-3) in the blood vessels, kidneys, heart, nerves, and other organs due to a missing enzyme.

Migalastat treats Fabry disease by increasing the activity of the deficient or the missing enzyme; thereby reduces the build-up of the particular type of fat in the body. The most common side effects experienced by patients during migalastat therapy are headache, fever, nausea, urinary tract infection, nasal and throat irritation. During the treatment with migalastat patients showed a more significant reduction in the level of globotriaosylceramide (GL-3) in the blood vessels of the kidneys.

Mogamulizumab-kpkc

Poteligeo (Mogamulizumab-kpkc) injection for intravenous use is used to treat two rare types of non-Hodgkin lymphoma such as Mycosis fungoides and Sézary syndrome in adult patients who have already received one systemic therapy. Mogamulizumab-kpkc is a monoclonal antibody which binds to a specific protein found on cancer cells.

Mogamulizumab-kpkc is the first FDA approved drug mainly to treat Sézary syndrome and provides a new treatment option for treating patients with Mycosis fungoides. The serious side effects of mogamulizumab-kpkc are skin toxicity, infusion-related reactions, muscle pain, loose stools, autoimmune problems, and infections of the upper respiratory tract. The infusion-related reactions caused by Mogamulizumab-kpkc injection can be minimized by premedicating patients with fever-reducing medications and drugs to treat allergies.

Patisiran

Onpattro (Patisiran) injection for intravenous use is used to treat polyneuropathy, a most common form of neuropathy in which the nerves outside the brain and the spinal cord are damaged. It is used in adult patients with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR). Patisiran blocks the production of an abnormal form of protein transthyretin (TTR) resulting in the accumulation of amyloid deposits in peripheral nerves. This condition leads to loss of sensation, pain or immobility in the hands and legs.

Amyloid deposits also affect the functioning of vital organs such as kidneys, heart, eyes and gastrointestinal tract. Patients treated with patisiran can perform their daily activities without any trouble. The most common side effects of patisiran are headache, stomach pain, breathing difficulty, nausea and rapid reddening of the skin.

Segesterone acetate and ethinyl estradiol vaginal system

Annovera (Segesterone acetate and ethinyl estradiol vaginal system) is a combined hormonal contraceptive device for women of reproductive potential to prevent pregnancy. This device is the first FDA approved vaginal ring contraceptive that can be used for an entire year. Annovera is a reusable donut-shaped ring, non-biodegradable, and a flexible device which does not require refrigeration and can be stored at room temperature.

Segesterone acetate and ethinyl estradiol vaginal system should be placed in the vagina for three weeks and must be removed for one week during menstrual periods. This schedule should be followed for one year which would be approximately thirteen 28-day menstrual cycles. The most common side effects of this vaginal system are headache, nausea, painful menstruation, and itching of the genital area. Women should avoid smoking while using this device as it may increase the risk of serious heart-related side effects.

Stiripentol

Diacomit (Stiripentol) is used as an add-on therapy to treat seizures associated with Dravet syndrome, a rare form of epilepsy in patients 2 years of age and older whose seizures were not adequately controlled by clobazam and valproate. The dose of stiripentol is calculated based on a patient's body weight given in two or three divided doses.

The treatment with stiripentol should not be stopped suddenly as it may increase the risk of seizure frequency and status epilepticus. So, gradual withdrawal of stiripentol is advised. Consumption of alcohol or medications that suppresses the actions of the brain should be avoided to minimize the risk of sleepiness. Operating heavy machinery or driving is not advised as stiripentol may affect mental alertness. Patients and caregivers should consult their physician immediately if any behavioral changes such as suicidal thoughts or mood disorders occur.

September

Cemiplimab-rwlc

Libtayo (Cemiplimab-rwlc) injection for intravenous use was approved by FDA for treating patients with cutaneous squamous cell carcinoma, a specific advanced skin cancer. Cemiplimab-rwlc is used in patients whose cancer cells spread to other body parts and patients who cannot be treated by surgery or radiation. The recommended dose of cemiplimab-rwlc is 350 mg given directly into the vein for at least 30 minutes every three weeks.

The most common side effects of cemiplimab-rwlc injection are rashes, diarrhea, and infusion-related reactions. Cemiplimab-rwlc should not be given to women who are pregnant or breastfeeding. The treatment can be continued until the progression of the disease or when the treatment with cemiplimab-rwlc does not result in intolerable toxicity.

Dacomitinib

FDA approved Vizimpro (Dacomitinib) tablets for oral use as a first-line treatment for patients whose non-small cell lung cancer (NSCLC) moved to other body parts along with a specific mutation which is confirmed by an FDA-approved test. The prescribed dose of dacomitinib is 45 mg taken by mouth once daily. The dose can be reduced if any severe side-effects occur.

Pregnant or breastfeeding women should not take dacomitinib tablets. The common side effects of dacomitinib are loose stools, rashes, loss of appetite, weight loss, hair loss, itching, cough, nail disease, swelling and redness inside the mouth along with painful sores. Lung function can be affected by dacomitinib; therefore do not use in patients with interstitial lung disease (ILD).

Duvelisib

Copiktra (Duvelisib) capsules for oral use was approved by FDA to treat resistant chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) and follicular lymphoma (FL) in adults who were already treated with at least two therapies. The recommended dose of duvelisib is 25 mg to be taken twice daily.

Women who are pregnant and breastfeeding mothers should avoid taking duvelisib capsules. The most common side effects of duvelisib are liver damage, low white blood cell count or neutropenia, fever, tiredness, nausea, respiratory infection, and bone or muscle pain. Periodic blood cell counts and evaluating liver function should be done at regular intervals, and patients must be advised to consult their doctor if their cough worsens.

Fremanezumab-vfrm

Ajovy (Fremanezumab-vfrm) is used as a preventive treatment for a migraine headache in adult patients. Fremanezumab-vfrm comes as a prefilled syringe which should be given as a subcutaneous injection just under the skin either 225 mg monthly or 675 mg for every three months.

The most common side effects of fremanezumab-vfrm include injection site reactions (pain, swelling, and itching) and allergic reactions such as breathing difficulty and swelling of the tongue, face or the throat. Steroid treatment can be advised for patients reported with allergies. The treatment can even be discontinued if the allergic reactions are severe or life-threatening. Caution is required when using fremanezumab-vfrm in pregnant or breastfeeding women and patients with a history of severe liver or kidney disease.

Galcanezumab-gnlm

Emgality (Galcanezumab-gnlm) injection for subcutaneous use was approved by FDA for the preventive treatment of migraine in adults. It comes as single dose prefilled syringe which can be self-administered by the patient in the areas of abdomen, thigh, upper arm but not into the tender, bruised, or red skin.

Patients who take galcanezumab-gnlm injection must be advised to consult the doctor immediately if any symptoms and signs of allergic reactions occur. The most common side effects are pain, swelling, rashes, redness, and itching at the site of injection. Some patients may also experience breathing difficulty.

Moxetumomab pasudotox-tdfk

Lumoxiti (Moxetumomab pasudotox-tdfk) injection for intravenous use was approved by FDA for treating hairy cell leukemia (HCL) in adult patients whose cancer returns after treatment. Moxetumomab pasudotox-tdfk is used in patients who have been already treated with at least two cancer chemotherapy. HCL is a rare but a slow-growing form of cancer of the blood in which the bone marrow produces excess B cells or lymphocytes resulting in the decreased level of platelets, red blood cells, and white blood cells.

The most common side effects of moxetumomab pasudotox-tdfk are nausea, weakness, headache, accumulation of excess fluid in the body, fever, loose stools, anemia, and infusion-related reactions. Patients treated with moxetumomab pasudotox-tdfk must be carefully watched for any side effects such as electrolyte abnormalities or kidney damage and should be supported with adequate fluid maintenance. Breastfeeding during the treatment is not advised.

October

Baloxavir marboxil

FDA approved Baloxavir marboxil (Xofluza) tablets for treating flu or acute uncomplicated influenza in patients who are 12 years of age and above. Baloxavir marboxil should be taken as a single dose within 48 hours once the symptoms are observed. The recommended dose is 40 mg for patients who weigh between 40 kg to 79 kg and 80 mg for patients who weigh 80 kg and above.

Baloxavir marboxil tablets should not be taken along with dairy products, calcium-rich beverages, antacids, and oral supplements containing iron, selenium, zinc, or magnesium. The most common side effects of taking baloxavir marboxil tablets are loose stools and bronchitis. Do not recommend baloxavir marboxil for other infections except flu.

Elapegademase-lvlr

Revcovi (Elapegademase-lvlr) injection for intramuscular use was approved by FDA as an enzyme replacement therapy to treat adenosine deaminase severe combined immune deficiency (ADA-SCID) in children and adults. ADA-SCID is an inherited rare genetic disorder caused due to the lack of adenosine deaminase enzyme. This disorder mainly affects the infants and young children.

Patients with ADA-SCID have very low immunity and must be protected against infections until there is an improvement in the immune function. Caution is required while giving an elapegademase-lvlr injection to patients with low platelet count or thrombocytopenia.

Inotersen

FDA approved Tegsedi (Inotersen) subcutaneous injection to treat stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). The recommended dose of inotersen is 284 mg given as a subcutaneous injection once a week. The sites for injection are the upper thigh, abdomen, and outer area of the upper arm.

The treatment with inotersen must be initiated only under the medical supervision and patients should be advised to receive the injection on the same day each week. Patients should be monitored for their kidney and liver function during the treatment with inotersen. Inotersen should not be used in children below 18 years of age and patients with low platelet count or acute glomerulonephritis.

Omadacycline

Nuzyra (Omadacycline) injection and tablets are approved by FDA to treat community-acquired bacterial pneumonia (CABP) and acute bacterial skin and skin structure infections. Omadacycline is a broad spectrum antibiotic belongs to the tetracycline group antibiotics. Omadacycline is designed to act against anaerobes, Gram-positive, Gram-negative, atypical and certain drug-resistant strains.

Omadacycline should not be given to pregnant and breastfeeding women as it may result in birth abnormalities such as permanent teeth discoloration and enamel hypoplasia. The most common side effects of omadacycline are vomiting, nausea, elevated blood pressure, diarrhea, headache, inability to sleep and injection site reactions.

Sarecycline

FDA approved Seysara (Sarecycline) tablets to treat inflammatory lesions of non-nodular moderate to severe acne vulgaris in patients who are aged nine years and above. Sarecycline is a tetracycline derived antibiotic which should not be used to treat other infections. It must not be used for more than 12 weeks.

Sarecycline should not be given to pregnant mothers as it may cause abnormalities to the unborn babies. It may also cause permanent teeth discoloration. The most common side effect of sarecycline is nausea. It can also cause fertility problems in males. Do not stop taking or skip the dose of sarecycline tablets without the consent of the doctor.

Talazoparib

Talzenna(Talazoparib) capsules were approved to treat locally advanced breast cancer or whose breast cancer that has spread to other body parts. The recommended dose is 1 mg per day taken as a single dose by mouth. Do not take an additional dose of talazoparib if the patient vomits or misses a dose but to take the next scheduled dose as prescribed.

The most common side effects of talazoparib capsules were tiredness, vomiting, headache, hair loss (alopecia), diarrhea, decreased appetite, and low blood counts. Caution is required while treating patients with abnormal bone marrow function. The dose of talazoparib must be reduced while giving to patients with kidney damage. Talazoparib should not be given to pregnant or breastfeeding mothers.

November

Amifampridine

The U.S. Food and Drug Administration approved Amifampridine tablets for treating Lambert-Eaton myasthenic syndrome (LEMS) in adults, which is a rare autoimmune disorder that affects the connection between nerves and muscles causing weakness. This is the first FDA approved drug for LEMS.

The most common side effects experienced by patients were burning like sensation (paresthesia), respiratory tract infections, abdominal pain, nausea, headache, back pain, hypertension etc. Patients should inform their doctor immediately if they have signs of allergic reactions such as rash, itching, fever, swelling or trouble breathing. Liver tests should be monitored regularly.

Emapalumab-lzsg

The U.S. Food and Drug Administration approved emapalumab-lzsg for the treatment of pediatric (newborn and above) and adult patients suffering from primary hemophagocytic lymphohistiocytosis (HLH) who have a resistant, recurrent or progressive disease.

It is available as an injection to be given into the patient's vein. Recommended initial dosage is 1 mg/kg as an intravenous infusion over an hour twice a week. Dexamethasone should be given concomitantly with emapalumab.

Common side effects caused by emapalumab were infections, high blood pressure, infusion-related reactions, low potassium, and fever. Patients receiving emapalumab should not receive any live vaccines.

Giltertinib

The U.S. Food and Drug Administration approved Gilteritinib tablets for treating adult patients who have recurrent or refractory acute myeloid leukaemia (AML) with an FLT3 mutation detected by an FDA-approved test. The LeukoStrat CDx FLT3 Mutation Assay is used to detect the FLT3 mutation in patients with AML.

Common side effects reported were muscle pain, joint pain, fatigue and increased liver enzymes. Doctors are advised to monitor patients for posterior reversible encephalopathy syndrome (a disease characterized by headache, confusion, seizures(fits) and visual loss), prolonged QT interval (abnormal heart rhythm) and pancreatitis (inflammation in the pancreas).

Women who are pregnant or breastfeeding should not take giltertinib because it may cause harm to a developing baby.

Glasdegib

The U.S. Food and Drug Administration approved Glasdegib tablets to be used in combination with low-dose cytarabine (a medicine used for cancer) for treating newly-diagnosed blood cancer named acute myeloid leukaemia (AML) in adult patients who are above 75 years. Common side effects reported were low red blood cell count (anaemia), tiredness (fatigue), bleeding (haemorrhage) etc.

Glasdegib should not be used during pregnancy or while breastfeeding. Male patients are advised to use condoms with a pregnant partner or a female partner that could become pregnant during treatment and for at least 30 days after the last dose. Patients should not donate blood or blood products during treatment with Glasdegib. ECG monitoring should be done regularly.

Larotrectinib

The U.S. Food and Drug Administration granted approval to larotrectinib for treating adult and pediatric patients whose cancers have a specific biomarker related to neurotrophic receptor tyrosine kinase (NTRK). This drug is especially useful for patients who have no satisfactory alternative treatment.

Common side effects reported were fatigue, nausea, cough, constipation, diarrhea, dizziness, vomiting. Liver tests should be monitored every two weeks during the first month of treatment, then monthly and as clinically indicated. Women who are pregnant or breastfeeding should not take larotrectinib, as it causes harm to the developing baby. Patients should report any signs of neurologic reactions such as dizziness immediately to their doctor.

Lorlatinib

Lorlatinib tablets are approved by USFDA for the treatment of patients with non-small cell lung cancer (NSCLC) which has spread to other body parts. The recommended dose is 100 mg once daily to be taken by mouth. Most common adverse reactions are edema and peripheral neuropathy. Pregnant women are advised about the potential risk to the developing baby. Females of reproductive potential are advised to use an effective non-hormonal method of contraception since lorlatinib can make hormonal contraceptives ineffective. Patients are warned about the risk of serious liver damage.

Revefenacin

Revefenacin inhalation solution was approved by USFDA for the maintenance(long-lasting) treatment of patients with chronic obstructive pulmonary disease (COPD). It should not be used in acute(sudden and severe) COPD. The common side effects caused by revefenacin are cough, nasopharyngitis (sore throat), upper respiratory tract infection, and back pain. Caution is exercised in patients with severe liver damage while using revefenacin. Patients are instructed to be alert if they experience symptoms like eye pain, eye discomfort or blurring of vision and difficulty or pain during urination while taking revefenacin, as it cause worsening of narrow-angle glaucoma and urinary retention.

Rifamycin

Rifamycin is an antibacterial medicine used for the treatment of travelers’ diarrhea caused by non-invasive strains of Escherichia coli in adults. It should not be used in patients below 18 years of age and in patients with diarrhea-associated with fever or bloody stools or due to microorganisms other than noninvasive E. coli. Rifamycin is available as 194 mg tablets. The recommended dose for travelers' diarrhea is two tablets, two times a day taken by mouth for three days. Most common side effects are headache and constipation.

December

Calaspargase pegol-mknl

Calaspargase pegol-mknl is an enzyme used as a component of a multi-agent chemotherapeutic regimen for treating acute lymphoblastic leukemia (blood cancer) in patients aged one month to 21 years.
It is available as an injection to be given into the vein(intravenous).
It should not be given in patients with a history of severe hypersensitivity(allergic) reaction or pancreas damage or bleeding events to the previous pegylated L-asparaginase and patients with severe liver damage. The common side effects reported were elevated transaminase(liver enzyme) and bilirubin, pancreas damage, diarrhea, etc.

Prucalopride

USFDA approves prucalopride for treating chronic idiopathic constipation (CIC) in adults. It is available as 1 mg or 2 mg tablets to be taken by mouth (orally) once a day.
Prucalopride is contraindicated in patients with:
Hypersensitivity to prucalopride.
Intestinal perforation or obstruction of the gut wall, Crohn’s disease, ulcerative colitis, and toxic megacolon.
Common side effects of prucalopride are headache, abdominal pain, nausea, diarrhea, abdominal distension, dizziness, vomiting, flatulence, and fatigue.
Patients taking prucalopride should be monitored regularly for worsening of depression and the emergence of suicidal thoughts and behavior.

Ravulizumab

The U.S.F.D.A approved Ultomiris (Ravulizumab) injection for treating adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disease.
Common side effects reported by patients involved in clinical trials were headache and upper respiratory infection.
Caution is needed when giving ravulizumab in patients with systemic(whole body) infections.
Meningococcal vaccine should be given at least two weeks before administering the first dose of ravulizumab.
Ravulizumab received Orphan Drug designation which provides incentives to assist and foster the development of drugs for rare diseases.

Tagraxofusp-erzs

Tagraxofusp-erzs injection is approved by USFDA for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients aged above 2 years.
There is a chance for liver damage. Patients should be monitored for liver enzymes regularly.
Most common side effects reported with tagraxofusp-erzs are capillary leak syndrome, nausea, fatigue, peripheral edema, pyrexia(high temperature) and weight gain.
Most common laboratory abnormalities are decreased albumin, platelets, hemoglobin, calcium, sodium, and increased glucose.