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Timber Pharmaceuticals Receives Positive Opinion On Orphan Designation From European Medicines Agency For TMB-001, Expected To Issue Its Decision On Orphan Designation In ~30 Days

European Commission expected to issue its decision on orphan designation in approximately 30 days BASKING RIDGE, NJ, Sept. 14, 2022 (GLOBE NEWSWIRE) -- via NewMediaWire -- Timber Pharmaceuticals, Inc.

Benzinga · 09/14/2022 08:09

European Commission expected to issue its decision on orphan designation in approximately 30 days

BASKING RIDGE, NJ, Sept. 14, 2022 (GLOBE NEWSWIRE) -- via NewMediaWire -- Timber Pharmaceuticals, Inc. ("Timber" or the "Company") (NYSE:TMBR), a clinical-stage biopharmaceutical company focused on the development and commercialization of treatments for rare and orphan dermatologic diseases, today announced that the Committee for Orphan Medicinal Products (COMP) at the European Medicines Agency (EMA) issued a positive opinion on the Company's application for orphan designation for TMB-001 for the treatment of autosomal recessive congenital ichthyosis (ARCI). Based on the COMP's opinion, the European Commission is expected to issue its decision on the orphan designation in approximately 30 days.

"We are pleased that the EMA's COMP has issued a positive opinion for orphan designation for TMB-001, recognizing the significant unmet medical need in the ARCI community for a safe and effective treatment option," said John Koconis, Chairman and Chief Executive Officer of Timber. "This is a first and important step towards market approval in Europe and encouraging as we work to advance TMB-001 in our ongoing pivotal Phase 3 clinical trial as rapidly as possible. I want to thank our team at Timber and all trial investigators and patients who have helped us reach this stage in development. As we wait for the European Commission to issue its decision to hopefully grant orphan designation for TMB-001 in ARCI, Timber now plans to also submit an application for EMA orphan designation for TMB-001 for the treatment of X-linked ichthyosis."

The EMA's orphan designation is available to companies developing treatments for rare diseases that are life-threatening or chronically debilitating that affect fewer than five in 10,000 people across the European Union (EU). Medicines that are granted orphan designation by the European Commission qualify for financial and regulatory incentives including protocol assistance at reduced fees during product development, access to centralized marketing authorization, and 10 years of marketing exclusivity in the EU after product approval.

TMB-001 is a topical isotretinoin, formulated using the Company's patented IPEG™ delivery system, in development for the treatment of moderate to severe forms of congenital ichthyosis (CI) including ARCI and X-linked recessive ichthyosis (XLRI). CI is a group of rare genetic keratinization disorders that lead to dry, thickened, and scaling skin. In patients with ARCI and XLRI, cutaneous manifestations include large, dark scaling throughout the body. There are currently no FDA-approved treatments for CI. TMB-001 was previously granted breakthrough therapy designation and fast track status from the U.S. Food and Drug Administration (FDA) for the treatment of CI.

Timber is currently assessing the efficacy, pharmacokinetics and safety of TMB-001 (0.05% isotretinoin) in the ongoing pivotal Phase 3 ASCEND clinical trial at leading research centers in the U.S., Canada, Italy, France, and Germany. Timber expects to enroll approximately 142 patients with moderate to severe CI in this study.

About Timber Pharmaceuticals, Inc.

Timber Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of treatments for rare and orphan dermatologic diseases. The Company's investigational therapies have proven mechanisms-of-action backed by decades of clinical experience and well-established CMC (chemistry, manufacturing, and control) and safety profiles. The Company is initially focused on developing non-systemic treatments for rare dermatologic diseases including congenital ichthyosis (CI) and sclerotic skin diseases. For more information, visit www.timberpharma.com.