Dive Brief:
- Both Sarepta and Biomarin are developing drugs for the rare pediatric disease Duchenne muscular dystrophy (DMD). Each company's drug has received the FDA's rare pediatric disease designation.
- DMD is a muscle degeneration disease, which affects mainly male children and adolescents. Maximum life expectancy is generally early 20's.
- Despite numerous setbacks along the way, both drugs are up for FDA review within the next eight months.
Dive Insight:
On June 10, when the FDA issued guidelines on acceptable endpoints and trial design for DMD candidate drugs, it was a major breakthrough. Patients, advocates and healthcare providers had rallied long and hard to allow Sarepta and Biomarin, to test their respective drugs, eteplirsen and drisapersen, in a smaller pool of patients, with a modified trial design. The challenge had been that the trials were small, a normal byproduct of testing a rare disease, and the endpoints needed to be more flexible.
Both drugs are up for approval and each company could also get a prized priority review voucher. Priority review vouchers have steadily increased in value since BioMarin sold its coupon to Regeneron and Sanofi last year for $67.5 million. That voucher was used to expedite approval of the PSCK9 inhibitor, Praluent.
Priority review vouchers grant companies the right to a six-month FDA review process, versus the standard 10-month process. Since last year, there have been several sales of vouchers, each with an increasing value. The latest sale happened just last week when AbbVie bought a priority review voucher from United Therapeutics for $350 million.