Xenpozyme (olipudase alfa) Approved by European Commission as First and Only Therapy for Acid Sphingomyelinase Deficiency (ASMD)
Sanofi
Pharmaceutical R&D Developer
European Commission
The European Commission, abbreviated as the EU Commission, is a supranational body under the European Union. Within the EU political system, the European Commission primarily undertakes executive tasks, thus being roughly equivalent to the government in a national system. However, the European Commission has other functions as well. In particular, except for the few circumstances specified in the treaties, the European Commission is the only institution with legislative power in the EU legislative process.
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News on June 29, 2022 /BioValleyBIOON/ -- Sanofi recently announced that the European Commission (EC) has approvedXenpozyme (olipudase alfa): This drug is an enzyme replacement therapy (ERT) used for patients with type A/B or type B acid sphingomyelinase deficiency (ASMD).ChildrenAnd adult patients, treating the non-central nervous system (non-CNS) manifestations of ASMD.
ASMD is a rare, progressive, and potentially life-threatening disease.Xenpozyme is the first approved therapy for the treatment of ASMD. In clinical trials, Xenpozyme treatment effectively improved various non-central nervous system clinical manifestations in pediatric and adult patients with ASMD.
This EU approval is based on data from the ASCEND and ASCEND-Peds clinical trials. These trials confirmed that, in adult and pediatric patients with ASMD,Xenpozyme treatment demonstrated strong clinically relevant improvements in lung function (measured by diffusing capacity of the lungs for carbon monoxide [DLco]), as well as reductions in spleen and liver volumes., with good tolerability and safety.
ASMD, historically known as Niemann-Pick disease types A, A/B, and B, is a rare, progressive, and potentially life-threatening genetic disorder. ASMD represents a spectrum of disease, with two types that may represent the extremes of a continuous disease spectrum: ASMD type A and ASMD type B. ASMD type A/B is an intermediate form that includes varying degrees of central nervous system (CNS) involvement.
Xenpozyme (olipudase alfa) is an enzyme replacement therapy (ERT) designed to replace the deficient or defective acid sphingomyelinase (ASM),This is an enzyme that allows sphingomyelin to break down.The accumulation of sphingomyelin in cells can cause damage to the lungs, spleen, liver, and other organs, potentially leading to early death. Currently, Xenpozyme is being studied in pediatric and adult patients to treat the non-CNS manifestations of ASMD. Xenpozyme has not been studied in patients with ASMD type A.
In the EU, the United States, and Japan, olipudase alfa has been grantedPriority Medicines (PRIME), Breakthrough Therapy Designation (BTD), SAKIGAKE Designation (Innovative Drugs).
In March 2022, Xenpozyme received approval in Japan through SAKIGAKE (a priority pathway for the review and approval of new drugs in Japan), marking the first regulatory approval of olipudase alfa worldwide. In the United States,FDAThe review period for the olipudase alfa Biologics License Application (BLA) has been extended by 3 months, and a new target action date (PDUFA date) has been set for October 3, 2022. (Bioon.com)
Source of the original text:Xenpozyme (olipudase alfa) approved by European Commission as first and only treatment for ASMD