BioMarin acquired Kyndrisa when it bought Prosensa of Leiden, Netherlands, in November 2014 for $680 million. In addition to the cash, there were two $80 million milestone payments related to regulatory approvals in the U.S. and Europe, which Prosensa shareholders will now not receive.
BioMarin’s abandonment of Kyndrisa once again puts the focus on Sarepta Therapeutics (SRPT). That company’s DMD drug, eteplirsen, has been on a very dramatic roller-coaster ride. Its FDA review was postponed in January until April due to a pending snowstorm on the East Coast. In February, 109 members of Congress sent a letter to the FDA urging it to accelerate approval of a DMD drug, not specifying any drug, but suggesting it should approve any treatment, and soon.
In March, 36 DMD experts signed a letter to the FDA urging approval of Sarepta’s eteplirsen. It was written by Carrie Miceli and Stanley Nelson, co-directors of the Center for Duchenne Muscular Dystrophy at UCLA.
In April, the FDA held a hearing that was a bit of a media circus. It went on for 12 hours and had 52 public commentators, often offering emotional cries for help, including several boys with DMD and their parents. The final decision was to be made on May 26, but on May 25 the FDA and Sarepta announced that the FDA was going to miss the deadline because “they are continuing their review and internal discussions related to our pending NDA for eteplirsen.”
Some people feel that BioMarin’s announcement today will place some pressure on the FDA to approve Sarepta’s eteplirsen.
“There’s only one standing now,” said Christine McSherry, executive director of the Kingston, Massachusetts-based Jett Foundation, to the San Francisco Business Times. McSherry is also the mother of a 20-year-old Duchenne patient, Jett, who was involved in clinical trials of both drugs. “There would seem to be some sideways pressure (on the FDA) to approve” eteplirsen.
Jett McSherry received three doses of drisapersen, but had a reaction at the injection site. Upon receiving Sarepta’s eteplirsen, he was stable after nine weeks and, according to Christine McSherry, he recovered some fine motor skills. “Honestly,” she told The San Francisco Business Times, “I think the drug saved his life.”
BioMarin has indicated that it still expects its 2017 adjusted profit to break even at the very least, but had previously indicated that it might need to write off all or most of the costs tied to the Prosensa acquisition. The company has also indicated the change may result in layoffs, although how many, when and where has not been indicated.