Chiesi Global Rare Diseases Announces FDA Acceptance of BLA Filing for Velmanase Alfa for the Proposed Treatment of Alpha-Mannosidosis

- FDA grants Priority Review designation with PDUFA date in the first half of 2023 -

BOSTON, Sept. 12, 2022 /PRNewswire/ -- Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), announced today that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) and granted Priority Review designation for velmanase alfa, the company's investigational enzyme replacement therapy for the proposed treatment of alpha-mannosidosis. The FDA set an action date in the first half of 2023 under the Prescription Drug User Fee Act (PDUFA).

"People who are living with rare diseases can count on our long-term commitment to research and innovation as evidenced by the progress we have made in advancing our development program in alpha-mannosidosis to a BLA filing," said Giacomo Chiesi, Head of Chiesi Global Rare Diseases. "Alpha-mannosidosis is an under recognized and progressive disease, and there may be many patients who are undiagnosed and untreated. The current management of alpha-mannosidosis is largely focused on treatment of the various symptoms. We believe that our investigational enzyme replacement therapy has the potential to address the cause of the disease and look forward to working closely with the FDA throughout its review process."

Alpha-mannosidosis is an ultra-rare genetic disorder that begins in childhood and progresses through adulthood. It is characterized by a deficiency of the enzyme alpha-mannosidase that results in the body's cells being unable to properly break down certain groups of complex sugars. The buildup of sugars can affect many parts of the body's organs and systems including the central nervous system. Effects of the disease vary significantly from person to person and progress over time. Symptoms may change as a patient gets older and can include recurrent chest and ear infections, hearing loss, distinctive facial features, muscle weakness, skeletal and joint abnormalities, visual abnormalities, or cognitive abnormalities. The prevalence of alpha-mannosidosis is approximately 1-9/1,000,000 worldwide.

"As someone who knows personally how devastating alpha-mannosidosis can be, this is an important milestone toward a potential first treatment option that addresses the root cause of the disease and is bringing patients, parents, families and caregivers hope for a better future," said Mark Stark, treasurer at the International Society for Mannosidosis & Related Disorders (ISMRD) and father of a son living with alpha-mannosidosis. "ISMRD is pleased to be collaborating with Chiesi in their efforts to address a serious and significant unmet medical need in the alpha-mannosidosis community and look forward to further updates on their clinical development program."

Velmanase alfa is a recombinant form of human alpha-mannosidase intended to provide or supplement natural alpha-mannosidase, an enzyme that helps with the degradation of mannose–rich oligosaccharides to prevent their accumulation in various tissues in the body. In 2018, Chiesi Group received marketing authorisation from the European Commission for Lamzede® (velmanase alfa) for the treatment of non-neurological manifestations in patients with mild to moderate alpha‑mannosidosis.

Priority Review is granted to therapies that the FDA determines have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. This designation shortens the FDA review period following the acceptance of the BLA compared to a standard review.

Chiesi Global Rare Diseases is a business unit of the Chiesi Group established in February 2020 and focused on research and development of treatments for rare and ultra-rare disorders. The Global Rare Diseases unit works in collaboration with Chiesi Group to harness the full resources and capabilities of our global network to bring innovative new treatment options to people living with rare diseases, many of whom have limited or no treatments available. The unit is also a dedicated partner with global leaders in patient advocacy, research and patient care. For more information visit www.chiesirarediseases.com.

Based in Parma, Italy, Chiesi is an international research-focused pharmaceuticals and healthcare group with over 85 years' experience, operating in 30 countries with more than 6,000 employees (Chiesi Group). To achieve its mission of improving people's quality of life by acting responsibly towards society and the environment, the Group researches, develops and markets innovative therapeutic solutions in its three focus areas: AIR (products and services that promote respiration, from new-born to adult populations), RARE (treatment for patients with rare and ultra-rare diseases) and CARE (products and services that support specialty care and consumer-facing self-care). The Group's Research and Development centre is based in Parma and works alongside 6 other important research and development hubs in France, the U.S., Canada, China, the UK, and Sweden to pursue its pre-clinical, clinical, and regulatory programmes. In 2018 Chiesi has changed its legal status to a Benefit Corporation, according to the law in Italy, USA and, more recently, in France, by incorporating common benefit objectives into its bylaws, to generate value for its business, for the society and the environment. Since 2019, Chiesi has been the world's largest B Corp certified pharmaceutical group. B Corps are global leaders convinced to leverage business as a force for good. Moreover, as a Benefit Corporation, Chiesi Farmaceutici S.p.A. is required by law to report annually in a transparent way about its progress in achieving the common benefits objectives it has set forward. The Group is committed to becoming carbon neutral by the end of 2035.

For more information, please visit www.chiesi.com.

Chiesi Global Rare Diseases Media Contact
Jenna Urban
Berry & Company Public Relations
1-212-253-8881
[email protected]

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SOURCE Chiesi Global Rare Diseases