RARE Daily

FDA Grants M6P Therapeutics Six Rare Pediatric Disease designations for LSDs

January 28, 2021

Rare Daily Staff

The U.S. Food and Drug Administration granted six Rare Pediatric Disease designations to M6P Therapeutics for various programs in its development pipeline for lysosomal storage disorders, including four recombinant enzyme and two gene therapy programs.

In addition, the FDA granted two Orphan Drug designations for its gene therapy programs for Gaucher disease and mucolipidosis.

Lysosomal storage diseases (LSDs) are a group of approximately 50 rare genetic disorders that are characterized by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies. The disorders can present in infancy, childhood, or adulthood. Although the signs and symptoms vary, in each case symptoms occur because an enzyme deficiency inhibits the ability of the lysosomes present in each of the body’s cells to break down complex components into simpler ones. When this process does not take place, the substrate begins to accumulate in the cells. The symptoms of LSDs are generally progressive over a period of time. Currently, there are no cures for any of these disorders.

M6P Therapeutics is developing an innovative technology that regulates the natural mechanism for trafficking enzymes to lysosomes. The company’s bicistronic-S1S3 platform enables improved bio-distribution of recombinant enzymes to target tissues and efficient cross-correction for gene therapies.

“LSDs are progressive and represent significant burden for patients, their families, and the healthcare systems,” said Pawel Krysiak, president and CEO of M6P Therapeutics. “Our innovative platform enables efficient trafficking of either a recombinant enzyme or gene therapy product to the affected cells and tissues and has the potential to generate best-in-class treatments for LSDs.”

The FDA grants rare pediatric disease designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. The designation makes each of M6P’s six experimental therapeutics eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Rhythm Pharmaceutics sold its priority review voucher for $100 million in January 2021 to Alexion Pharmaceuticals.

Orphan Drug designation serves to advance drug development for drugs or biologics that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions that affect fewer than 200,000 Americans. Orphan designation provides development and commercial incentives including exemption from FDA user fees and eligibility for a seven-year period of market exclusivity upon approval in the United States.

Photo: Pawel Krysiak, president and CEO of M6P Therapeutics

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