Minovia Therapeutics has received orphan drug designation from the US Food and Drug Administration (FDA) for its investigational mitochondrial cell therapy MNV-201 in myelodysplastic syndrome (MDS).

The new status adds to the therapy’s existing FDA Fast Track designation in MDS and complements earlier designations for Pearson syndrome, including Fast Track and rare paediatric disease status.

The Israeli biotech said the latest designation strengthens regulatory support for MNV-201 as it continues clinical testing of what it describes as a first-in-class cell therapy designed to restore mitochondrial function.

Natalie Yivgi-Ohana, co-founder and CEO of Minovia, said: “We continue to receive validation from the FDA for the potential of our lead product, MNV-201, this time in the form of orphan drug designation in MDS. MNV-201 targets the mitochondria, a critical multi-functional organelle. FDA designations such as ODD underscore the urgency of drugs treating these diseases affecting smaller populations, while providing additional benefits across the FDA process that, we expect, will prove both medically and financially valuable.”

Chief scientific officer Noa Sher added: “Orphan drug designation for MNV-201 marks an important milestone in our mission to address critical challenges in mitochondrial health in both primary and acquired mitochondrial diseases. By leveraging our expertise in mitochondrial and haematopoietic science, and through the innovative mechanism of action of our drug product, we hope to bring forward a treatment option that could significantly improve outcomes for MDS patients.”

Orphan drug designation provides regulatory incentives including tax credits for clinical testing, fee reductions and seven years of market exclusivity following approval. The status is granted to therapies targeting rare conditions affecting fewer than 200,000 people in the US.

MDS is a blood disorder characterised by ineffective haematopoiesis, cytopenia and an elevated risk of progression to acute myeloid leukaemia (AML). Minovia’s research suggests MDS may be an age-related mitochondrial disease, and the company is conducting a Phase 1b study of MNV-201 in low-risk MDS patients, with six of nine participants dosed so far.

MNV-201 uses the company’s proprietary Mitochondrial Augmentation Technology (MAT) to add healthy, energy-producing mitochondria into a patient’s own stem cells, aiming to restore organ function and improve overall health. Early studies in Pearson syndrome showed a strong safety profile and evidence of multi-system benefit, including improved muscle and blood function.

Separately, Minovia has announced a definitive business combination agreement with Launch One Acquisition Corp, a Nasdaq-listed special purpose acquisition company. The transaction, expected to close later in 2025, will result in the combined entity operating as Minovia Therapeutics under a new ticker symbol.