Niemann-Pick Disease Drug Type C Treatment Market, by Drug Type (Phase III Drug {Trappsol Cyclo, IB1001}, Marketed Drug {Miglustat (Zavesca)}), by Indication (Niemann-Pick Disease Drug Type C1, Niemann-Pick Disease Drug Type C2), by Distribution Channel (Hospital Pharmacies, Online Pharmacies, Retail Pharmacies), and by Region (North America, Latin America, Europe, Asia Pacific, Middle East, And Africa) Size, Share, Outlook, and Opportunity Analysis, 2022 - 2030

Published In : Dec 2022

Code : CMI4275

Pages :362

Formats :

Excel and PDF

Industry : Pharmaceutical

Market Challenges And Opportunities

Global Niemann-Pick Disease Drug Type C Treatment Market – Driver

Increasing number of company collaborations regarding niemann-pick disease drug type C treatment by market players is expected to fuel the market growth over the forecast period. For instance, in March 2020, Evox Therapeutics Limited, a leading exosome therapeutics company, announced the signing of a rare disease-focused partnership with Takeda Pharmaceutical Company Limited, a multinational pharmaceutical company. The collaboration will focus on developing up to five novel protein replacement and mRNA therapies, including Evox's preclinical programme in Niemann-Pick disease type C (NPC) and a second new programme directed at another undisclosed rare disease

Increasing number of product approvals is expected to boost the global niemann-pick disease drug type C treatment market growth over the forecast period. For instance, in March 2022, Azafaros B.V., a company developing new treatment options to help patients with rare metabolic disorders such as lysosomal storage diseases (LSDs), announced that the U.S. Food and Drug Administration (FDA) had granted Orphan Drug Designation (ODD) for AZ-3102, a novel small molecule with a unique dual mode of action, in Niemann-pick disease type C (NP-C). The designation was based on promising preclinical data of AZ-3120 in a NP-C mouse model.

Global Niemann-Pick Disease Drug Type C Treatment Market-Restraint

Termination in clinical trials is expected to hamper the growth of global niemann-pick disease drug type C treatment market over forecast period. For instance, in April 2022, Mandos, LLC, a biopharmaceutical company, terminated a phase II/III trial in Niemann-pick disease type C (In children, adolescents, and adults) in Australia, France, Germany, Italy, New Zealand, Singapore, Spain, Turkey, the U.K. and the U.S. (Intrathecal) (NCT02534844) (EudraCT2015-002548-15). The U.S. Food and Drug Administration (FDA) did not find the drug to be safe and effective for treating Niemann-pick disease type C.