Ionis Pharmaceuticals, Inc. (IONS - Free Report) announced that its partner, Biogen Inc. (BIIB - Free Report) has completed the rolling submission of a New Drug Application (NDA) for nusinersen in the U.S. for the treatment of spinal muscular atrophy (SMA). In addition, Biogen has requested the FDA for Priority Review of the candidate.
The submission was based on results from an interim analysis of the phase III ENDEAR study, which demonstrated that infants receiving nusinersen experienced a statistically significant improvement in the achievement of motor milestones.
A Marketing Authorization Application (MAA) for nusinersen is expected to be filed in the EU in the coming weeks. Regulatory filings in other countries are expected in the upcoming months.
Note that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has granted Accelerated Assessment to the candidate.
Moreover, nusinersen enjoys Orphan Drug designation in both the U.S. and the EU. In the U.S., the candidate has been granted Fast Track status as well.
SMA is estimated to affect about 30,000–35,000 patients in the U.S., the EU and Japan. In the U.S., one in every 50 individuals, which means approximately six million people, carries the gene mutation that causes SMA.
With no currently approved drugs for this indication, the SMA market presents high potential with significant unmet needs.
Investors’ focus should remain on regulatory updates on nusinersen at the company, going forward.
Both Ionis and Biogen carry a Zacks Rank #3 (Hold).
Stocks to Consider
A couple of better-ranked stocks in the health care sector are Corcept Therapeutics Incorporated (CORT - Free Report) and Anika Therapeutics, Inc. (ANIK - Free Report) . Both the stocks sport a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Corcept Therapeutics witnessed an increase of 25% and 12.5% in its earnings estimates for 2016 and 2017, respectively, in the last 60 days. The company has posted a positive earnings surprise thrice in the last four quarters, bringing the average beat to 100.00%. The company’s share price has surged 39.5% year to date.
Meanwhile, Anika Therapeutics’ earnings estimates for 2016 and 2017 were up a respective 12.6% and 14% over the last 60 days. The company’s earnings have outpaced estimates in each of the last four quarters with an average beat of 42.19%. The company’s share price has jumped 20.3% year to date.
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