Niemann-Pick disease type C (NPC) is a lysosomal storage disorder that doesn't happen very often. NPC makes cholesterol and other lipids build up in different parts of the body, including the brain. Different people show different NPC signs at different times. Mostly, symptoms start in youth and get worse over time, causing problems that can be life-threatening. NPC is caused by changes in the NPC 1 and NPC 2 genes, which are passed down in a way called "autosomal recessive." Over the projection period, market growth is anticipated to be fueled by the rising number of medication type C collaborations amongst industry players treating Niemann-Pick disease. Over the projection period, rising product approvals are anticipated to fuel expansion in the Niemann-Pick disease medication type C market.
The global Niemann-Pick disease drug type C treatment market is anticipated to grow at a CAGR of 22.0% during the period between 2022 and 2030.
Major Players in the Niemann-Pick Disease Drug Type C Treatment Industry:
1. Johnson & Johnson
Johnson & Johnson is an American global firm that was established in 1886 and produces consumer packaged goods, medications, and medical equipment. It was founded in 1886 and headquartered in New Jersey, U.S. It operates in 60 countries.
2. Intrabio
Intrabio was founded in 2015 and headquartered in Oxfordshire, United Kingdom. It operates in all European countries. In Oct 2020, IntraBio provides additional information regarding the positive results of the multinational clinical trial IB1001 for the treatment of Niemann-Pick disease type C.
3. Cyclo Therapeutics, Inc.
Cyclo Therapeutics, Inc. is a clinical-stage biotechnology company that focuses on drug development using cyclodextrins. It was founded in 1990 and headquartered in Florida, United States. It operates in U.S and other 79 countries. In Mar 2023, Cyclo Therapeutics provides an update on its business, which includes the Pivotal Phase 3 global study (TransportNPCTM) evaluating Trappsol® CycloTM for Niemann-Pick Disease Type C1 (NPC1), which is expected to enroll 40% of participants by the end of March.
4. Kempharm, Inc.
Kempharm, Inc. was founded in 2006 and headquartered in Iowa, United States. It operates in multiple countries. The name of KemPharm, Inc. has been changed to Zevra Therapeutics, Inc. On March 1, 2023, the Company began trading under the new ticker symbol "ZVRA" on The Nasdaq Global Market. In Feb 2023, Zevra Therapeutics, Inc. said that arimoclomol, the company's oral, first-in-class investigational product candidate being developed as a treatment for Niemann-Pick disease type C (NPC), will be featured in two poster presentations at the 19th Annual WORLDSymposiumTM 2023, the annual research conference for lysosomal diseases that will be held in February 2023 in Orlando, Florida, USA.
5. Azafaros B.V.
Azafaros B.V aims at developing new therapeutic agents for the treatment of rare metabolic disorders. It was founded in 2018 and headquartered in Zuid-Holland, The Netherlands. It operates in 21 countries. In June 2023, Azafaros is working on a clinical trial for AZ-3102 to treat Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C. The trial is in Phase I, which is the first step toward approval.
6. StrideBio.
StrideBio focuses on making and developing new technologies based on adeno-associated viral (AAV) vectors. It was founded in and headquartered in North Carolina, United States. It operates in 9 countries. In Dec 2019, Sarepeta Therapeutics and StrideBio will collaborate to develop novel gene therapies for four neurological genetic disorders: MECP2 (Rett syndrome), SCN1A (Dravet syndrome), UBE3A (Angelman syndrome), and NPC1 (Niemann-Pick).
7. Sarepta Therapeutics, Inc.
Sarepta Therapeutics, Inc. is a global biopharmaceutical company, was founded in 1980 and headquartered in Massachusetts, United States. It operates across the world.
*Definition: Niemann-Pick disease type C (NPC) is a slowly progressive lysosomal disorder whose principal manifestations are age dependent
