Yesterday, the US Food and Drug Administration (FDA) approved Sarepta’s Exondys 51 (eteplirsen), the first approved treatment for Duchenne Muscular Dystrophy (DMD).
The product - a once weekly intravenous infusion of 30 milligrams per kilogram - is a major breakthrough for the disease which affects around one in every 3,500 – 5,000 males born worldwide.
In a conference call to discuss the achievement, Sarepta’s CFO Sandy Mahatme said the drug would be available in a 2ML and 10 ML vial, costing $1,600 and $8,000 respectively.
He also told stakeholders the firm has “sufficient supply and capacity to supply the US market for over a year” and has various initiatives underway to expand capacity to supply both the US, and other markets clinically and – potentially – commercially.
One of these initiatives is the sale of a rare pediatric disease priority review voucher (PRV) which the firm also received from the FDA yesterday.
“We can use this voucher to receive priority review on one of our own future market applications that would otherwise be a standard review or we can transfer the voucher to another company as a source of non-dilutive financing,” Mahatme said.
And, he added, the firm is considering the latter, exploring a sale of the PRV in order “to offset some of the costs associated with the continued build out of our clinical development pipeline, our manufacturing scale up, and entry into European markets.”
Currently the firm uses various manufacturers in the US but is looking to bring on “backup manufacturers” as the drug progresses in Europe. “Simultaneously,” said Mahatme, “we will expand from what we have right now, which is midscale manufacturing capacity to a larger scale.”
Priority Review Voucher
The FDA’s priority review voucher system was proposed in 2006 as an incentive for drug companies to target neglected tropical diseases, and was later spun out to include rare paediatric diseases. Holders of such vouchers are eligible for a six month expedited review for their New Drug Application (NDA).
Sarepta becomes one of just a dozen or so recipients to have received a PRV, and could benefit from a substantial fee if it decides to sell.
In 2014, Knight Therapeutics sold its neglected tropical disease PRV to Gilead for $125m which subsequently used it to expedite review of its HIV drug Odefsey - a combination of three drugs: Edurant (rilpivirine) and Emtriva (emtricitabine) and tenofovir alafenamide.
And last year, United Therapeutics sold its rare paediatric disease PRV to AbbVie for $350m, though AbbVie is yet to have disclosed ow it intends to use it.