FDA Fast Tracks Rare Disease Treatment

Article

Elamipretide targets the inner mitochondrial membrane and helps preserve mitochondrial energetics in patients with primary mitochondrial myopathy.

The FDA has granted Fast Track designation for a drug candidate designed to help patients suffering from primary mitochondrial myopathy.

Stealth BioTherapeutics developed the drug elamipretide, formerly known as Bendavia, in hopes to treat this rare genetic disorder that impairs health with muscle weakness and severe fatigue. Currently there are no FDA approved medications.

Researchers from the Institut de recherches cliniques de Montréal (IRCM) discovered a family of molecules, elamipretide being among them.

“Elamipretide targets the inner mitochondrial membrane and helps preserve mitochondrial energetics,” said drug discoverer Peter W. Schiller, PhD. “Due to this property, elamipretide could become the first treatment designed for primary mitochondrial myopathy. The FDA’s grant of a Fast Track designation for this treatment is therefore excellent news.”

The FDA’s Fast Track program increases the likelihood for eligibility of priority review and accelerated approval if certain criteria is met for these drugs that treat serious conditions.

Related Videos
male pharmacist using digital tablet during inventory in pharmacy | Image Credit: sofiko14 - stock.adobe.com
Pharmacist holding medicine box in pharmacy drugstore. | Image Credit: I Viewfinder - stock.adobe.com
Pharmacy Drugstore Checkout Cashier Counter | Image Credit: Gorodenkoff - stock.adobe.com
Medicine tablets on counting tray with counting spatula at pharmacy | Image Credit: sutlafk - stock.adobe.com
Capsules medicine and white medicine bottles on table | Image Credit: Satawat - stock.adobe.com
Human cell or Embryonic stem cell microscope background | Image Credit: Anusorn - stock.adobe.com
Concept of health care, pharmaceutical business, drug prices, pharmacy, medicine and economics | Image Credit: Oleg - stock.adobe.com
Biosimilar pharmaceutical drug bottle on blue background. | Image Credit: Carl - stock.adobe.com
© 2024 MJH Life Sciences

All rights reserved.