FDA grants breakthrough designation to Timber’s congenital ichthyosis candidate

The FDA has granted a breakthrough designation to Timber Pharmaceuticals’ topical isotretinoin formulation for the treatment of congenital ichthyosis, the company announced in a press release.

TMB-001, a topical formulation using Timber’s patented IPEG delivery system, is being developed for the treatment of moderate to severe forms of congenital ichthyosis (CI) including X-linked recessive ichthyosis and autosomal recessive CI lamellar ichthyosis.

“This is a significant moment for people who are living with CI, their families and caregivers, and clinicians and researchers who have been working for years to find new treatment options for this debilitating condition,” John Koconis, chairman and CEO of Timber, said in the release. “A breakthrough therapy designation is no small achievement.”

According to Koconis, 16 breakthrough applications were rejected by the FDA or withdrawn during the first half of FDA’s 2022 fiscal year, while nine applications were granted.

“I am proud of our team for demonstrating the potential of TMB-001 in our phase 2b program, and we are rapidly pushing forward with a pivotal phase 3 clinical trial,” Koconis said.

The phase 2b CONTROL study showed a clinically meaningful reduction in CI severity with TMB-001 and a phase 3 ASCEND trial has begun.

A breakthrough therapy designation allows development and review to be expedited for drugs that could treat serious or life-threatening conditions.