- Sarepta on Monday finally got approval from the U.S. Food and Drug Administration for the first Duchenne muscular dystrophy (DMD) drug, naming it Exondys 51 (eteplirsen).
- The biotech will charge approximately $300,000 annually per patient. The price is based on the weight of the patient. Exondys 51 will be available in both a 2 ml and 10 ml vial. The 2 ml vial will cost $1,600 and the 10 ml vial will cost $8,000.
- The recommended dose of Exondys 51 is 30 mg/kg, administered once weekly, as a 35 minute to 60 minute intravenous infusion. The drug has no contraindications, but can only be used in DMD boys who have the faulty exon 51 gene.
Sarepta was lauded by analysts for pricing Exondys at $300,000. While that might seem like an expensive treatment, it is low relative to other rare disease drugs on the market. The population that is eligible for treatment with the drug is very small — just a few thousand boys.
"We did take into consideration how much R&D was spent prior to this time. Obviously we have a large number of post-marketing requirements that we are doing now and we'll continue to do. And we're committed to continuing to do research in the following exons. We're committed to looking at our next -generation chemistry and so we've really included all of this in our pricing considerations," said Sarepta Chief Medical Officer and Interim CEO Edward Kaye during a call with analysts after the market closed on September 19.
"And given the sensitivity to pricing, we've tried to be very — we think is reasonable given all of the costs for this, and even the innovation that goes into manufacturing," he added.
The company intends to immediately begin education of physicians about the importance of genetic testing to determine if a patient has the exon 51 mutation. Sarepta has also set up therapeutic specialists and account managers around the country to begin engaging payers.
"We have a very experienced national accounts team that will be calling on payers across the country. As is typical for most rare disease launches, we anticipate to take 30 days to 90 days for patients to get coverage. So, we expect a lag of this duration before any sales are recognized," said Sarepta's head of global development Bob Cumbo.
The company has also set up a patient assistance program dubbed SareptAssist that will provide patients with dedicated case managers and give financial assistance, as well as help with claims processing.
"Commercially, our team is in place and our supply chain is ready to provide product to patients," added Cumbo. Sarepta said that it has enough supply currently to serve patients for over a year.
The biotech plans to help pay for post-marketing commitments and manufacturing with the sale of its priority review voucher, obtained from the FDA to help aid the review process of other pediatric rare disease drugs. These vouchers can be sold to other companies to expedite review of any pending drug.