Sarepta is offering its Duchenne therapy Exondys 51 in certain countries outside of the U.S. through a compassionate use program managed by Clinigen, as the company reported better-than-expected revenues from the med in the U.S. and as the EMA is still evaluating the drug in due process.
The EMA accepted Sarepta’s application for Exondys 51 last December, and the company is in a six-month “clock stop” period to collect additional data, with a review decision from the EMA’s Committee for Medicinal Products for Human Use expected by the first half of 2018.
The drug’s approval by the FDA last September stirred up controversy because of what some sees as a lack of clinical data to confirm the drug’s effectiveness. But the Massachusetts biotech managed a net revenue of $35 million for the second quarter of this year from sales of Exondys 51 in the U.S., exceeding the Wall Street’s previous estimate of around $22 million.
During his first earnings call on Wednesday since taking the helm at Sarepta less than a month ago, Douglas Ingram described the results as very positive, and it also promoted Sarepta to raise its whole-year guidance from over $95 million in revenue to between $125 million and $130 million.
The announcement came close on the heels with what Sarepta sees as a favorable patent dispute settlement it had reached with BioMarin Pharmaceutical on exon-skipping products. The agreement requires Sarepta to make a one-time payment of $35 million plus additional milestones to BioMarin. But it also gives Sarepta “broad global freedom to operate for our exon-skipping compounds … it permits us to confidently execute our worldwide strategic plans,” Ingram said during the earnings call. “And it allows us to focus our time and our resources not on legal issues but rather on launching our therapies worldwide and continuing to develop our deep pipeline to the best of our patients around the world.”
Ingram said he expects the managed access program will start to turn in moderate revenues in the fourth quarter, though the company did not include that in its revenue outlook adjustment.
“The MAP program is extremely important because it gives the providers the opportunity to find additional patients early, pre EMA approval, and get access to the drug, get used to the drug, see the benefits of the drug,” said Ed Kaye, the stepping-down CEO who remains on Sarepta’s board, during the earnings call. “These programs, while wonderful for the patients to get early access, it really gives a lot of the providers confidence in understanding how to prescribe it, so when the drug does get reimbursed through the health technology assessment groups, then you typically have a much faster launch.”
The program is currently planned for 13 countries, but will be expanded to others later. Sarepta would not share information on the drug’s price in the EU, saying that physicians will receive it confidentially after they submitted compassionate use requests for the drug for eligible patients.