- 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.
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.