French drugmaker Sanofi announced that the US Food and Drug Administration (FDA) has granted priority review for its enzyme replacement therapy used in the treatment of Pompe disease.
Specifically, the FDA accepted the priority review of Sanofi’s (SNYNF) Biologics License Application (BLA) for the enzyme replacement therapy, avalglucosidase alfa. The FDA’s deadline for announcing a decision is May 18, 2021.
The long-term therapy is for the treatment of Pompe disease, which is a rare, degenerative muscle disorder caused by a genetic deficiency or dysfunction of the lysosomal enzyme GAA, that can affect an individual’s ability to move and breathe, the company said. The disease affects an estimated 3,500 people in the US and can break out at any age from infancy to late adulthood.
Avalglucosidase alfa is an investigational enzyme replacement therapy designed to improve the delivery of GAA enzyme to muscle cells, and if approved, would offer a potential new standard of care for patients with Pompe disease, Sanofi stated.
“The hallmarks of Pompe disease are the relentless and debilitating deterioration of the muscles, which causes decreased respiratory function and mobility,” said Sanofi’s Karin Knobe. “Avalglucosidase alfa is specifically designed to deliver more GAA enzyme into the lysosomes of the muscle cells. We have been greatly encouraged by positive clinical trial results in patients with late-onset and infantile-onset Pompe disease.”
Sanofi said that the BLA submission is supported by positive data from two clinical trials. Avalglucosidase alfa has already received FDA breakthrough-therapy and fast-track designations for the treatment of patients with Pompe disease.
In October, the European Medicines Agency accepted the marketing authorization application for avalglucosidase alfa as a long-term enzyme replacement therapy in the treatment of patients with Pompe disease for review. Meanwhile, the Medicines and Healthcare Products Regulatory Agency in the UK has granted promising innovative medicine designation for avalglucosidase alfa.
Shares of Sanofi have advanced 12% over the past year and are up less than 1% since the beginning of the year. Looking ahead, the average analyst price target of $121.51 reflects about 22% upside potential in the shares in the coming 12 months.
Earlier this month, Sanofi made an offer to snap up biotech company Kiadis for an aggregate adjusted equity value of €308 million ($358.5 million), in a move to expand its range of cell-based immunotherapy products.
Commenting on the deal, Mizuho analyst Mara Goldstein said that the acquisition highlights the corporate interest in this area of biotech.
“The terms of the acquisition appear more advantageous to Sanofi than funding associated with the previous collaboration, but nonetheless suggest to us that there is corporate appetite for cell therapy development,” Goldstein wrote in a note to investors.
The vast majority of Wall Street analysts are bullish about the stock. The Strong Buy consensus is based on 9 Buy ratings versus 3 Hold ratings. (See Sanofi stock analysis on TipRanks)
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