RARE Daily

FDA Grants Fast Track Designation to Translate Bio’s mRNA Therapy for Cystic Fibrosis

February 26, 2020

Rare Daily Staff

The U.S. Food and Drug Administration has granted Fast Track designation to Translate Bio’s experimental mRNA therapeutic, MRT5005, for the treatment of cystic fibrosis. 

Cystic fibrosis (CF) is a rare, life-shortening genetic disease. It is caused by mutations in the CFTR gene that lead to a defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) protein. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating non-working or too few CFTR proteins at the cell surface. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

MRT5005 is the first inhaled mRNA therapeutic with delivery to the lungs, designed to address the underlying cause of cystic fibrosis, regardless of genetic mutation, and address the underlying cause of CF, regardless of genetic mutation. It delivers mRNA encoding fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to cells in the lung through nebulization. The phase 1/2 clinical trial of MRT5005 is currently ongoing with results expected to be reported in the third quarter of 2020.

“While there have been significant advances in the treatment of CF, substantial unmet need remains. Roughly 10 percent of the CF population is not anticipated to benefit from currently approved CFTR modulators,” said Ann Barbier, chief medical officer, Translate Bio. “MRT5005 has the potential to treat all people with CF, including those with mutations that result in limited to no CFTR protein production. The Fast Track designation will help Translate Bio to expedite the clinical development of this potentially transformative therapeutic.”

The FDA’s Fast Track program facilitates the expedited development and review of drugs that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs. Clinical development programs granted Fast Track designation may be eligible for several benefits, including more frequent meetings and communications with the FDA and, if relevant criteria are met, the potential for Accelerated Approval, Priority Review and Rolling Review of a Biologics License Application. The FDA has also granted MRT5005 the Orphan Drug designation.

Photo: Ann Barbier, chief medical officer, Translate Bio

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