Azafaros Receives Additional Regulatory Designations for AZ-3102 from FDA, EMA and MHRA

Leiden, The Netherlands, February 20, 2023 – Azafaros B.V. today announced its progress in ongoing interactions with Health Authorities regarding its lead asset, AZ-3102.

In the last two weeks, Azafaros has been granted the following designations:

  • Two Rare Pediatric Disease Designations (RPDD) by the United States Food and Drug Administration (FDA) for the treatment of GM1 and GM2 gangliosidoses
  • Orphan Medicinal Product Designation (OMPD) by the European Medicines Agency (EMA) for the treatment of GM2 gangliosidosis
  • An Innovation Passport by the UK Medicines and Healthcare Products Regulatory Agency (MHRA) for the treatment of GM1 and GM2 gangliosidoses


“The designations by the FDA, EU, and UK regulatory authorities allow us to accelerate the development of our lead program, AZ-3102, as a potential novel disease-modifying approach for these highly underserved lysosomal storage disorders with neurological involvement that often affect patients at a very young age,” said Stefano Portolano, Chief Executive Officer of Azafaros.

The designations have been granted based on the rarity of the diseases, the high unmet medical need and promising preclinical data in in vivo models of primary and secondary gangliosidoses in which AZ-3102 treatment showed prolonged survival and improved motor function. The results add to the proof-of-concept of AZ-3102’s unique mode of action and highlight its disease-modifying potential in GM1 and GM2.

The FDA’s RPDD and Voucher Program[1] is intended to facilitate the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases (RPD), defined as indications affecting fewer than 200,000 people in the US in which severe or life-threatening manifestations primarily affect individuals aged from birth to 18 years. Companies that receive marketing approval in a RPD may be eligible to receive a voucher for priority review of a subsequent marketing application for a different product. The voucher may be used by the company or can be sold to a third party.

OMPD[2] is granted by the EMA for the development of therapeutics for rare diseases which affect fewer than 5 in 10,000 people across the EU. Medicines that meet the EMA’s orphan designation criteria qualify for financial and regulatory incentives that include a 10-year period of marketing exclusivity in the EU after product approval, protocol assistance from the EMA at reduced fees during the product development phase, and access to the centralized marketing authorization procedure.

The Innovation Passport provides entry to the UK’s Innovative Licensing and Access Pathway (ILAP)[3], which aims to accelerate time to market and facilitate patient access to innovative medicines that address the needs of patients with life-threatening or seriously debilitating diseases.


About AZ-3102

AZ-3102 is an orally available azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NP-C).

In 2022, the compound received Fast Track Designation for GM1 and GM2 gangliosidoses as well as Niemann-Pick disease Type C (NP-C), and Orphan Drug Designations (ODD) for GM2 gangliosidosis (Sandhoff and Tay-Sachs Diseases) and NP-C from the FDA.


About GM1 and GM2 Gangliosidoses

GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) are lysosomal storage disorders caused by the accumulation of GM1 or GM2 gangliosides, respectively, in the central nervous system (CNS), resulting in progressive and severe neurological impairment and early death. These diseases mostly affect infants and children, and no disease-modifying treatments are currently available.


About Niemann-Pick Disease Type C (NP-C)

Niemann-Pick disease type C (NP-C) is a progressive, life-limiting neurological lysosomal storage disorder caused by mutations in the NPC1 or NPC2 gene and aberrant endosomal-lysosomal trafficking, leading to the accumulation of various lipids, including gangliosides in the CNS. The onset of disease happens throughout the lifespan of an affected individual, from prenatal life through adulthood. The mainstay of therapy is symptom management.


About Azafaros

Azafaros is a clinical stage company founded in 2018 with a deep understanding of rare genetic disease mechanisms, a compound library from Leiden University, and led by a team of highly experienced industry experts. Azafaros aims to build a pipeline of disease-modifying therapeutics to offer patients and their families new treatment options. The company’s lead clinical-staged program is AZ-3102 a small molecule, orally available, brain penetrant azasugar, with the potential to treat GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) and Niemann-Pick disease type C (NP-C). By applying its know-how, network, and courage, the Azafaros team challenges traditional development pathways to rapidly bring new drugs to the rare disease patients who need them. Azafaros is supported by a syndicate of leading Dutch and Swiss investors including Forbion, BioGeneration Ventures, BioMedPartners and Schroders Capital.

For media information:

Azafaros B.V.

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