Early access program launched for muscular dystrophy medicine

Pharma IQ News

A biotech firm is providing eligible patients suffering from a severe muscle degeneration disease fast-track access to a brand new treatment.

Sarepta Therapeutics Clinigen's Idis Managed Access division is initiating a managed access program for eteplirsen in certain geographies. The product will be available to eligible Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping - a method use to restore genes from mutations.

Data from clinical studies of the eteplirsen injection in a small number of DMD patients demonstrated a consistent safety and tolerability profile. However, the full clinical benefits of eteplirsen is not established yet.

DMD is a rare degenerative neuromuscular disorder causing severe progressive muscle loss and premature death that impacts 3,500-5000 males worldwide. Muscle weakness in the lower limbs spreads to the arms, neck and other areas of the body. Death occurs before the age of 30 generally due to respiratory or cardiac failure.

“Patients with a particular type of Duchenne muscular dystrophy will now have access to an approved treatment for this rare and devastating disease.” said Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research after the authority granted the treatment with accelerated approval.  

She added: “In rare diseases, new drug development is especially challenging due to the small numbers of people affected by each disease and the lack of medical understanding of many disorders. Accelerated approval makes this drug available to patients based on initial data, but we eagerly await learning more about the efficacy of this drug through a confirmatory clinical trial that the company must conduct after approval.”

Although there are plans to expand the reach of the project over time, it is initially running in Europe, North America and South America.

Requests to enter the program must be entered by a physician on behalf of a patient.

These programs are used to enable physicians to legally provide patients that fit required medical criteria with access to treatment that is not yet approved. Clinigen delivers over 220 Managed Access Programs to thousands of patients.

Early Access Program resources:

Early Access Programs: EU Compliance Handbook 

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