Orvida Pharma Announces FDA Acceptance of KM-023 (ontivatrep) into Rare Disease Evidence Principles Process for Olmsted Syndrome

FDA’s Rare Disease Evidence Principles (RDEP) process aims to provide greater speed and predictability in the review of therapies for rare diseases with significant unmet medical need

Acceptance enables engagement with FDA on the data needed to substantiate the safety and effectiveness of ontivatrep

Topline results from Phase 1b clinical trial in patients with Olmsted syndrome anticipated around year-end 2026

NESS ZIONA, Israel, Oct. 14, 2026 (GLOBE NEWSWIRE) -- Orvida Pharma, a clinical-stage biotechnology company developing first- and best-in-class treatments for rare and severe genetic skin diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted KM-023 (ontivatrep), the Company’s lead oral investigational therapy for Olmsted syndrome, into the FDA’s Rare Disease Evidence Principles (RDEP) process.

“Acceptance into the FDA’s Rare Disease Evidence Principles process represents an important regulatory milestone for Orvida and the development of ontivatrep for Olmsted syndrome,” said David Aviezer, PhD, MBA, Chief Executive Officer of Orvida Pharma. “The process provides an opportunity to engage with the FDA on the evidence needed to support potential approval, an important consideration in developing treatments for ultra-rare diseases with significant unmet medical need. We look forward to working closely with the Agency as we advance ontivatrep.”

The RDEP process is intended to provide greater clarity and predictability regarding the types of evidence that can be used to demonstrate substantial evidence of effectiveness for eligible rare diseases. Accepted sponsors meet with the appropriate FDA review team to determine the data needed to substantiate safety and effectiveness. Depending on the circumstances, this may include one adequate and well-controlled study together with robust confirmatory evidence, which may include mechanistic or biomarker evidence, findings from relevant nonclinical models, clinical pharmacodynamic data, case reports, expanded access data or natural history studies.

Ontivatrep is a highly specific and selective oral TRPV3 inhibitor being developed for Olmsted syndrome, severe keratodermas and ichthyosis. Olmsted syndrome is a rare and severe genetic skin disorder characterized by painful, thickened skin, particularly on the palms and soles, that can significantly impair mobility and quality of life and, in some cases, can be life-threatening. There are currently no approved therapies for Olmsted syndrome.

In April 2026, Orvida reported favorable Phase 1a safety data for ontivatrep from a randomized, placebo-controlled study in 30 healthy volunteers. The Company is currently conducting a Phase 1b clinical trial in patients with Olmsted syndrome in Europe and the United States. Topline results are expected around year-end 2026.

Orvida will deliver a corporate presentation during BIO-Europe 2026, taking place November 9–11 in Cologne, Germany, and participate in the Jefferies Global Healthcare Conference in London, November 16–19. The Company will be available for one-on-one meetings with investors and pharmaceutical companies at both conferences.

Ontivatrep is a proposed generic name for KM-023. The International Nonproprietary Name (INN) and United States Adopted Name (USAN) for ontivatrep are pending.

About Orvida Pharma

Orvida Pharma is a clinical-stage biotechnology company developing first- and best-in-class treatments for rare and severe genetic skin diseases. The Company’s lead investigational candidate, ontivatrep, is a novel, highly specific, and selective oral TRPV3 inhibitor being developed for the treatment of Olmsted syndrome, severe keratodermas and ichthyosis. Orvida’s management team comprises industry leaders with deep expertise in drug discovery, dermatological pharmaceutical development, and rare disease therapeutics.

Contact Information

Investors and Media
Marcy Nanus
Managing Partner
Trilon Advisors, LLC
mnanus@trilonadvisors.com