FDA Grants Timber Fast Track Designation for Rare Dermatologic Disease

The U.S. Food and Drug Administration granted Timber Pharmaceuticals Fast Track designation to TMB-001, the company’s experimental therapy for the rare dermatologic conditions X-linked recessive ichthyosis and autosomal recessive congenital ichthyosis lamellar ichthyosis.

Congenital Ichthyosis (CI) is a group of rare genetic keratinization disorders that lead to dry, thickened, and scaling skin. People living with CI may have limited range of motion, chronic itching, an inability to sweat normally, high risk of secondary infections, and impaired eyesight or hearing. CI affects about 80,000 people in the U.S. and more than 1.5 million globally.

TMB-001, a topical isotretinoin in Timber’s patented IPEG delivery system, is being developed for the treatment of moderate to severe subtypes of congenital ichthyosis. At the end of 2021, Timber announced positive top line results for its phase 2b CONTROL Study of TMB-001.

“Based on the clinical success that TMB-001 has shown to date, we believe we have an important opportunity to dramatically improve the lives of people living with congenital ichthyosis who currently have no FDA-approved treatments and limited standard of care options,” said John Koconis, chairman and CEO of Timber. “The designation of Fast Track status is a significant achievement that speaks to the unmet need in CI. Now we can communicate frequently with the FDA throughout our pivotal phase 3 ASCEND clinical trial with the goal of earlier drug approval and access by patients.”

Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The designation allows for more frequent meetings and written communication from the FDA to discuss a drug’s development plan and the design of proposed clinical trials to ensure collection of appropriate data needed to support drug approval.

Fast Track designation also allows FDA to conduct a rolling review of a marketing authorization application, which means that a drug company can submit completed sections of its application to FDA for review, rather than waiting until every section of the NDA is completed before the entire application can be reviewed. A drug that receives Fast Track designation may also be eligible for Accelerated Approval and Priority Review.

Author: Rare Daily Staff