FDA Grants Breakthrough Therapy Designation to Venglustat in the US, Sanofi (SNY) Reports

Sanofi (NASDAQ:SNY) is one of the best undervalued stocks under $50 to invest in now. Sanofi (NASDAQ:SNY) announced on March 18 that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to venglustat for the treatment of neurological manifestations of type 3 Gaucher disease, which is a rare lysosomal storage disorder. Venglustat is a novel, investigational oral glucosylceramide synthase inhibitor.

Management stated that the designation is based on data from the LEAP2MONO phase 3 study, in which patients who received venglustat exhibited  statistically significant improvements in neurological symptoms measured by a global test score that included assessments for ataxia and cognition compared with patients receiving the enzyme replacement therapy, imiglucerase. It reported that the venglustat was overall well tolerated in the study, with no new safety signals compared with previous studies.

Sanofi (NASDAQ:SNY) added that the most commonly reported adverse events included headache, nausea, diarrhea, and spleen enlargement. Venglustat previously attained fast-track designation from the FDA for its potential use in GD3, as well as orphan designation for GD3 in the US, EU, and Japan. Sanofi (NASDAQ:SNY) also stated that it will pursue global regulatory filings for venglustat in GD3 during 2026.

Sanofi (NASDAQ:SNY) researches, produces, and distributes pharmaceutical products. The company’s operations are divided into the Pharmaceuticals, Consumer Healthcare, and Vaccines segments.

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