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Conexiant chevron_right Dermatology chevron_right FDA Grants New EB Treatment Designation
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FDA Grants New EB Treatment Designation

Squamous cell carcinoma is the leading cause of mortality in severe epidermolysis bullosa," notes Dr. Robert Kirsner, as FDA grants rare pediatric designation to mitochondrial-targeting therapy.

Conexiant

The U.S. Food and Drug Administration has granted Rare Pediatric Disease Designation for BPM31510T, a topical therapy for epidermolysis bullosa. The biotechnilogy company BPGbio announced the designation during EB Awareness Week.

Epidermolysis bullosa (EB) affected approximately 1 in 20,000 pediatric patients in the United States. Clinical features included:

  • Skin blistering
  • Chronic wounds
  • Scarring
  • Squamous cell carcinoma (SCC) risk
  • Life-threatening complications.

The designation was the second Rare Pediatric Disease Designation for BPGbio's BPM31510 franchise, following a designation in September 2024 for primary coenzyme Q10 deficiency. The drug previously received U.S. Food and Drug Administration (FDA) Orphan Drug Designation.

Phase I clinical trial results demonstrated that BPM31510T was well tolerated as a topical therapy for EB, according to Robert S. Kirsner, MD, PhD, Chairman and Harvey Blank Professor of Dermatology at the University of Miami Miller School of Medicine and Vice President of the Wound Healing Society. The company announced plans for phase II/III trials in 2025.

Clinical trial observations included:

  • Completion of phase I trial
  • Phase II completion for squamous cell carcinoma
  • Plans for phase II/III trials.

BPM31510T targeted three wound-healing pathways:

  • Inflammation processes
  • Cell proliferation
  • Tissue remodeling.

The BPM31510 platform included multiple formulations:

Topical (BPM31510T):

  • Completed phase I trial for EB
  • Completed phase II for SCC
  • Phase II/III trials scheduled for 2025.

Intravenous (BPM31510IV):

  • Phase IIB trial for glioblastoma multiforme
  • Phase IIA completion for pancreatic cancer
  • Planned trial for CoQ10 deficiency mutations.

Oral Formulation:

  • Phase II trial planned for sarcopenia.

The development program proceeded with DEBRA of America, the U.S. EB patient advocacy organization. The Rare Pediatric Disease Designation included eligibility for priority review voucher upon approval.

BPGbio developed the treatment using their NAi Interrogative Biology Platform. The platform identified mitochondrial dysfunction in:

  • Epidermolysis bullosa
  • CoQ10 deficiency
  • Various cancers.

The company announced plans for trials at U.S. and EU sites.

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