RARE Daily

FDA Grants Rare Pediatric Disease Designation to Translate Bio’s mRNA Therapy for Cystic Fibrosis

April 1, 2020

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

Photo: Ann Barbier, chief medical officer, Translate Bio

Cystic fibrosis (CF) is a rare, life-shortening genetic disease 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 which lead to CF by creating non-working or too few CFTR proteins at the cell surface that 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. It delivers mRNA encoding fully functional cystic fibrosis 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.

As the worldwide healthcare system remains focused on the response to the COVID-19 pandemic, Translate Bio anticipates related interruptions in enrollment, dosing and follow-up in the ongoing phase 1/2 clinical trial in patients with CF. Translate Bio  originally expected to report results from the trial in the third quarter of 2020, but now will provide updated timing on the expected interim data readouts at a later date.

“As more patients with CF and their physicians choose to avoid unnecessary risk of exposure to SARS-CoV-2, we, like many companies across the healthcare and biopharmaceutical industry, anticipate that patient dosing and follow-up visits in our ongoing trial will be affected,” said Ann Barbier, chief medical officer, Translate Bio. “We are in regular communication with our clinical trial sites to monitor the impact of COVID-19 and to continue to provide the best possible care and support to the patients in the trial. Given the uncertain and dynamic nature of this situation, we will provide an update when we have additional insight into enrollment and dosing metrics.”

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes ARU1801 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, Swedish Orphan Biovitrum sold its Priority Review voucher to AstraZeneca for $95 million.

The FDA has also granted MRT5005 Orphan Drug and Fast Track designations.

Author: Rare Daily Staff

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