THE FDA HAS AWARDED FAST TRACK DESIGNATION TO TWO DRUGS CURRENTLY IN DEVELOPMENT FOR CMT:
ACCELERON’S ACE-083 AND PHARNEXT’S PXT3003
Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Fast Track addresses a broad range of serious conditions.
Fast Track designation must be requested by the drug company. The request can be initiated at any time during the drug development process. FDA will review the request and make a decision within sixty days based on whether the drug fills an unmet medical need in a serious condition.
Once a drug receives Fast Track designation, early and frequent communication between the FDA and a drug company is encouraged throughout the entire drug development and review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients.
SOURCE: U.S. FOOD AND DRUG ADMINISTRATION www.fda.gov/ForPatients/Approvals/Fast/ucm405399.htm
HNF partner, Acceleron Pharma, announced in November, 2018, that the FDA had granted Fast Track status to ACE-083, an injected myostatin drug intended to increase muscle mass and strength. Acceleron announced the exciting, preliminary positive results of the part 1 of the phase 2 clinical trial in January, 2019.
Currently, ACE-083 is recruiting patients with CMT Type 1 and CMT Type X in a part 2 of a phase 2 clinical trial.
“We’re pleased that the FDA has granted this designation for ACE-083,” said Robert K. Zeldin, M.D., Chief Medical Officer of Acceleron. “Patients with CMT currently have no approved therapies. To date, results from our Phase 2 trials have shown that patients treated with ACE-083 experience robust increases in muscle volume. If our ongoing clinical studies show that ACE-083 also improves functional outcomes and confirm the favorable safety profile observed thus far, the Fast Track process could help us work with the FDA to deliver it to patients as quickly as possible.”
Preliminary results from part 2 of the trials are expected by year end 2019.
HNF partner, Pharnext, announced in February, 2019, that they had been granted Fast Track designation for the development of PXT3003 for the treatment of patients with CharcotMarie-Tooth disease Type 1A (CMT1A). PXT3003 is a novel synergistic combination of baclofen, naltrexone and sorbitol, formulated as an oral solution that is given twice a day.
“We are pleased to receive Fast Track designation for PXT3003 in CMT1A” said Daniel Cohen, M.D., Ph.D., cofounder and Chief Executive Officer of Pharnext. “CMT1A is a rare, chronic neuropathy that affects at least 125,000 people across the U.S. and Europe. There is currently no approved treatment for this disease. We look forward to continuing our productive dialogue with the FDA to rapidly progress towards a U.S. New Drug Application for PXT3003, to deliver this therapy to patients as quickly as possible.”