The huge unmet medical need for a DMD treatment led several different companies to have a go at developing one, and in the last 12 months, several of them have gone under the FDA’s microscope.
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The FDA’s accelerated approval pathway, which expedites approval of drugs for treating serious or life-threatening diseases, can be based on data from adequate and well-controlled studies demonstrating the product has an effect on a surrogate endpoint “reasonably likely” to predict clinical benefit to patients’ comfort, quality of life, and/or survival, and provides an avenue for earlier patient access to promising new drug developments while the manufacturer conducts clinical trials created to verify predicted benefits.
Though growing awareness and earlier diagnosis of Duchenne muscular dystrophy could pressure the average price for Exondys, the firm also predicts the US market could expand beyond its 1,800 patient estimate.
Exondys 51 was also given an “orphan drug” designation, which provides certain financial incentives to drug makers to encourage the development of drugs for rare diseases. The most frequently reported adverse effects of the drug include balance disorder and vomiting.
“Today’s accelerated approval of EXONDYS 51 represents a major milestone in the treatment of Duchenne Muscular Dystrophy for patients amenable to skipping exon 51 by targeting the underlying genetic cause of the disease – the lack of the dystrophin protein”, said Edward Kaye, M.D., Sarepta’s interim chief executive officer and chief medical officer. The agency’s decision, however, showed the growing power of patients and advocates over evaluation of drugs.
The FDA also granted Sarepta a rare pediatric disease voucher representing a commitment by the FDA to review a new drug developed by Sarepta within six months rather than the standard 10 months or more.
This year, MDA already has committed more than $17 million to research, as it takes a unique big-picture perspective across the spectrum of muscle-debilitating diseases that take away everyday abilities such as walking, talking and hugging.
Sarepta Therapeutics shares spiked by as much as 86% in trading on Monday after the US Food and Drug Administration said it approved a key drug.
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Diana Zuckerman, president of the National Center for Health Research, a nonprofit research group, noted that the Exondys 51 clinical trial was poorly done. Other drugs are now being developed for patients who have different mutations. It is estimated that up to thirteen percent of people with DMD have mutations addressable by EXONDYS 51. Committee members voted 5-8 with no abstentions against accelerated approval of the drug, although Paul Romitti, professor in the University of Iowa’s department of epidemiology and interdisciplinary program in toxicology, later acknowledged that he meant to vote in favor of accelerated approval, despite a desire to see a “better controlled study”.
“These kids have hope now”, said Christine McSherry of Pembroke, whose 20-year-old son Jett benefitted from Sarepta’s treatment, called eteplirsen, in the clinical trial.
“It’s really hard for me to celebrate”, she said.
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Shares of Sarepta soared almost 80 percent on the news, trading at over $50 apiece early Monday. BioMarin Pharmaceuticals has since announced it was abandoning development of Kyndrisa.
