" class="no-js "lang="en-US"> Azafaros Receives Additional Regulatory Drug Designations
Thursday, March 23, 2023

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

Azafaros today announced its progress in ongoing interactions with Health Authorities regarding its lead drug 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 Program1 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.

OMPD2 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.

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