BioSpace News Archive

FDA

Timber Pharmaceuticals Receives FDA Breakthrough Therapy Designation for TMB-001 for the Treatment of Congenital Ichthyosis

Timber Pharmaceuticals, Inc. (“Timber” or the “Company”) (NYSE American: TMBR), a clinical-stage biopharmaceutical company focused on the development and commercialization of treatments for rare and orphan dermatologic diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to TMB-001, a topical isotretinoin formulated using the Company’s patented IPEG™ delivery system, for the treatment of congenital ichthyosis (CI).

May 31, 2022
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4 min read
Drug Development

Stealth BioTherapeutics Receives Orphan Drug Designation from the European Medicines Agency for Elamipretide for the Treatment of Myopathic Mitochondrial DNA Depletion Syndrome

Orphan drug designation applies to patients with primary mitochondrial myopathy due to nuclear DNA mutations (nPMM) in the NuPower Phase 3 clinical trial.

May 31, 2022
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6 min read