The Orphan Drug designation was created to aid and hasten the development of drugs created for the treatment, diagnosis, or prevention of rare diseases. When the FDA grants this designation, not only does it affirm that this treatment has the potential to help patients, but it brings other benefits as well. These include tax credits, exemptions from fees, and potential market exclusivity for seven years if approved.
Recently, an investigational treatment for Duchenne muscular dystrophy (DMD) was granted this designation. Titled elamipretide, the drug is developed by Stealth BioTherapeutics and is meant to offer a new and innovative option to DMD patients. Alongside granting this designation, the FDA also accepted Stealth’s request for a meeting before the submission of an Investigational New Drug (IND) application.
The purpose of this meeting will be to discuss the best way to move forward with elamipretide as an add-on to drugs in the therapeutic class of exon-skipping phosphorodiamidate morpholino oligomers (PMO) that have already received approval. Prior data has shown that the combination of these therapies leads to increased dystrophin expression in mouse models of DMD.
This is not the only pre-IND meeting that Stealth has had with the FDA. The company met with the Division of Cardiology and Nephrology in order to discuss the route to approval for cardiomyopathy associated with Duchenne’s.
You can read more about regulatory developments related to elamipretide at Yahoo! News.
DMD: A Quick Overview
Duchenne muscular dystrophy (DMD), is one of nine forms of muscular dystrophy. A mutation passed down in an X-linked recessive pattern stops patients from producing dystrophin, ultimately resulting in progressive muscle weakness and degeneration. Other symptoms include difficulty with motor skills, fatigue, learning disabilities, frequent falls, issues with moving positions, and difficulty walking. Complications may include heart disease and respiratory failure, making treatment integral. The main form of treatment is steroids, but there is ongoing research looking into new options for DMD. Other current options include amino acids, asthma treatment, physical and occupational therapy, heart medications, assisted breathing, carnitine, creatine, and other supplements.
