Until now, there have been no US-approved drugs to fight the disease; American sufferers only had steroids to slow loss of muscle strength.
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Company officials said the drug would cost about $300,000 per year.
People with DMD typically die in their 20s or 30s from the progressive disorder, which affects about one of every 3,600 males worldwide. The agency does not need to see proof that the drugs actually improve the patients’ ability to function or extend their lives before granting approval. Exondys 51 was approved to treat patients with a confirmed mutation of the DMD gene amenable to exon 51 skipping, which affects about 13 percent of the population with DMD.
Approval of Sarepta Therapeutics Inc.’s eteplirsen, announced Monday morning, marked the first treatment for the degenerative and often deadly disease Duchenne muscular dystrophy.
Exondys 51 doesnt cure Duchenne muscular dystrophy and will only help a minority of patients. Duchenne muscular dystrophy Duchenne muscular dystrophy is caused by lack of a functional dystrophin protein, a protein that helps keep muscle cells intact. While Sarepta didn’t have even a small study proving the drug’s benefit in a traditional sense, evidence that boys with the disease continued to walk for years after their diagnosis was significant, he said. Shares almost doubled in active trade Monday morning after the drug’s approval was announced – the stock move was only the fourth-largest in the company’s history – in contrast with previous setbacks, including an advisory committee’s recommendation against approving the drug earlier this year. Thus, the analyst believes that the increment in the price target is quite justified given the approval and a rewarding receipt that is confirmed as a priority review voucher.
After a comprehensive (and at times contentious) review process, the FDA has given Sarepta Therapeutics (NASDAQ: SRPT) a green light to begin marketing Exondys 51 to Duchenne muscular dystrophy, or DMD, patients.
Both FDA camps had “exercised reasonable scientific judgment”, Califf found, adding that it’s “exceedingly rare” to overrule a decision by the director of the Center for Drug Evaluation and Research. The FDA isn’t bound to follow the vote of these panels, but usually does. The news of Exondys 51’s approval sent shares in the company rocketing 74% today. Those scant data compared patients on eteplirsen to a “historical control”-previous data on patients deemed to have similar characteristics to those who took the drug-not to patients receiving a placebo in the study, which the FDA prefers”.
“Ultimately, it appears to us that the FDA bowed to external pressure from patient advocates and others who demanded that a safe and potentially efficacious drug be made available”, said Leerink Swann analyst Joseph Schwartz, in a note to investors. Needham & Company LLC increased their price objective on Sarepta Therapeutics from $47.00 to $81.00 and gave the company a “buy” rating in a research report on Tuesday. For these boys, who had previously faced certain death and no treatment options, any improvement in their condition was to be heralded, despite limited clincial data.
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Sarepta Therapeutics Inc (NASDAQ:SRPT) stock on Friday’s pre market session gained 4.50% at price of $9.52.
