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Sanofi's MAA for Pompe Disease Candidate Gets EMA's Acceptance

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Sanofi (SNY - Free Report) announced that the European Medicines Agency (EMA) has accepted its marketing authorization application (MAA) for review, seeking an approval for avalglucosidase alfa as a long-term enzyme replacement therapy (ERT) for the treatment of patients with Pompe disease, a rare, degenerative muscle disorder. A decision from the EMA is expected in the second half of 2021.

The MAA was based on positive data from two studies. Firstly, the pivotal phase III COMET study evaluated the safety and efficacy of avalglucosidase alfa in patients with late-onset Pompe disease compared to alglucosidase alfa (standard of care). Secondly, the phase II mini-COMET study investigated the safety and exploratory efficacy of avalglucosidase alfa in patients with infantile-onset Pompe disease, who were previously treated with the standard of care.

Per the company, an estimated 50,000 people are affected by Pompe disease worldwide. If approved, avalglucosidase alfa can become a potentially new standard of care for the given indication.

Shares of Sanofi have inched up 0.5% so far this year against the industry’s decrease of 1.7%.

We remind investors that in June 2020, Sanofi announced promising data from the COMET study evaluating avalglucosidase alfa for the treatment of late-onset Pompe disease. Avalglucosidase alfa met the primary endpoint of the study by demonstrating non-inferiority in improving respiratory function compared to the standard of care in Pompe disease.

Moreover, the patients treated with avalglucosidase alfa walked 30 meters farther than those treated with the standard of care as measured by the 6-minute walk test, a key secondary endpoint of the study.

The FDA already granted both Breakthrough Therapy and Fast Track designations to avalglucosidase alfa for the treatment of Pompe disease. The Medicines and Healthcare Products Regulatory Agency in the UK conferred a Promising Innovative Medicine status on avalglucosidase alfa to address the given indication.

Amicus Therapeutics (FOLD - Free Report) is developing its lead pipeline candidate AT-GAA, a differentiated biologic for Pompe disease. The company plans to initiate a rolling biologics license application (BLA) for AT-GAA later in 2020 to treat Pompe disease.

Zacks Rank & Stocks to Consider

Sanofi currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the large-cap pharma sector include Eli Lilly and Company (LLY - Free Report) and H. Lundbeck A/S (HLUYY - Free Report) , both presently carrying a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Eli Lilly’s earnings estimates have been revised 2.6% upward for 2020 and 2.2% for 2021 over the past 60 days. The stock has rallied 10.5% year to date.

H. Lundbeck’s earnings estimates have moved 16.4% north for 2020 and 8.8% for 2021 over the past 60 days.

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H Lundbeck AS (HLUYY) - free report >>