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Sanofi (SNY) Pompe Disease Drug Gets FDA's Priority Tag

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Sanofi (SNY - Free Report) announced that the FDA has granted priority review to its biologics license application (BLA) for its investigational enzyme replacement therapy (ERT), avalglucosidase alfa, for the treatment of Pompe disease, a rare degenerative muscle disorder. With the FDA granting a priority review, a decision is expected on May 18 next year.

The BLA was based on data from two pivotal studies, which include both infantile-onset and late-onset Pompe disease patients. This disease which causes a debilitating deterioration of the muscles resulting in decreased respiratory function and mobility, affects an estimated 3,500 people in the United States.

A similar application is under review in the EU with a decision expected in the second half of 2021.

Amicus Therapeutics (FOLD - Free Report) is also developing 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 with plans for completing the final submission in the first half of 2021.

So far this year, Sanofi’s shares have risen 0.6% against the industry’s 0.5% decline.


In a separate press release, Sanofi announced that the FDA has granted Fast Track designation to its investigational BTK inhibitor, rilzabrutinib for immune thrombocytopenia (ITP), a blood disorder that causes high risk for bleeding events. A phase III study on rilzabrutinib in persistent or chronic immune thrombocytopenia has been initiated recently. Rilzabrutinib was granted orphan drug designation by the FDA for ITP in October 2018.

Rilzabrutinib, which was added to Sanofi’s pipeline with this year’s acquisition of Principia Biopharma, is also being evaluated in late-stage studies for moderate-to-severe pemphigus, a rare skin disorder that causes lesions in the skin and in the mucous membranes. The candidate is also in phase II study for IgG4-Related Disease (IgG4-RD), a rheumatologic disease driven by chronic inflammation, immune cell infiltration and fibrosis within organs.

Novartis’ (NVS - Free Report) Promacta/Revolade and Amgen’s (AMGN - Free Report) Nplate are approved for treating chronic immune thrombocytopenia in patients who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.

In another press release, Sanofi announced that the European Commission has granted approval to its quadrivalent recombinant influenza vaccine, Supemtek for the prevention of influenza in adults. Supemtek contains three times more antigen than standard-dose vaccines, which coupled with the use of recombinant technology should provide improved protection against influenza.

Sanofi currently has a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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