Medindia LOGIN REGISTER
Medindia

Pompe Disease Market to Reach New Heights by 2030

by Dr. Jayashree Gopinath on Sep 9 2021 6:56 PM

A new report from GlobalData anticipates an increase in the Pompe disease market by 2030 through emergence of new drugs that target long term efficacy.

 Pompe Disease Market to Reach New Heights by 2030
An upcoming influx of new therapies into the Pompe disease (PD) pipeline in the next decade is expected to provide a significant boost to the treatment market.
Pompe disease is an inherited, and often fatal; disorder that can greatly damage the heart and skeletal muscles.In fact, the market will grow at an impressive compound annual growth rate (CAGR) of 13.5%.

Akash Patel, Pharma Analyst at GlobalData, comments: “The current PD pipeline is largely unconvincing to key opinion leaders (KOLs) interviewed by GlobalData, except for the possible addition of a gene therapy that may only require a single dose to be administered, which is an exciting prospect”.

This gene therapy has long-term efficacy and removes the need for a regular dosing schedule, which alone will have a huge impact on the lives of PD patients.

Although the long-term PD pipeline can be viewed as promising, they can be even more of powerful if these drugs addressed key needs such as restoring GAA enzyme function in the central nervous system (CNS) and preventing disease progression and damage in the long-term.

According to GlobalData’s report, ‘Pompe Disease – Opportunity Assessment and Forecast to 2030’, the PD market was worth $584.54m in 2020 in the three major markets (3MM*) and will grow to $1.95bn in 2030.

The PD pipeline in the 3MM has two therapies in late-stage development: Sanofi Genzyme’s avalglucosidase alfa, an enzyme replacement therapy (ERT) in pre-registration, and Amicus Therapeutics’ chaperone advanced ERT (CHART) cipaglucosidase alfa + miglustat in Phase III.

Advertisement
The overall clinical data from both therapies remains unconvincing to KOLs. In a direct head-to-head comparison with Myozyme, avalglucosidase alfa has not demonstrated a very significant improvement in efficacy.

There is currently only one therapy available in the PD market: Sanofi Genzyme’s Myozyme (agalsidase beta), which is available in both Germany and Japan and is marketed under the brand name Lumizyme in the US.

Advertisement
The patents on this ERT are expiring, and as a result, Sanofi Genzyme has developed avalglucosidase alfa to maintain its established share of the PD market.

Although this will provide access to another efficacious ERT for PD patients, an unmet need remains for therapies with novel mechanisms of action (MoAs) that significantly improve long-term outcomes.

Several gene therapies are currently in Phase I/II and expected to enter the US PD market in the next decade. These therapies are designed to be administered intravenously as a single-dose therapy and are also potentially curative to have long-term efficacy and do not require a regular dosing schedule.

In addition to late-stage pipeline agents acting as a driver for PD market growth, an increasing number of early diagnoses from the established new-born screening programs in the US and improvement in disease symptomatology from physicians will also contribute to steady growth of the PD market over the forecast period.

However, a potentially significant barrier to growth of the PD market will be the cost of therapies, leading to challenges around insurance coverage and reimbursement for novel therapies and particularly gene therapy.



Source-Medindia


Advertisement

Home

Consult

e-Book

Articles

News

Calculators

Drugs

Directories

Education