- Therapeutic Category : Fabry Diseases
- CAS Number : 108147-54-2
- Molecular Formula : C6H13NO4
- Molecular Weight : 163.17 g/mol
- Mesh Size : As per Customer Requirement
- Form : Capsule
- Metrochem Development Status : Commercial
Mechanism of Action:
Migalastat is a pharmacological chaperone that reversibly binds to the active site of the alpha-galactosidase A (alpha-Gal A) protein (encoded by the galactosidase alpha gene, GLA), which is deficient in Fabry disease. This binding stabilizes alpha-Gal A allowing its trafficking 6 Reference ID: 4305282 from the endoplasmic reticulum into the lysosome where it exerts its action. In the lysosome, at a lower pH and at a higher concentration of relevant substrates, migalastat dissociates from alpha-Gal A allowing it to break down the glycosphingolipids globotriaosylceramide (GL-3) and globotriaosylsphingosine (lyso-Gb3). Certain GLA variants (mutations) causing Fabry disease result in the production of abnormally folded and less stable forms of the alpha-Gal A protein which, however, retain enzymatic activity. Those GLA variants, referred to as amenable variants, produce alpha-Gal A proteins that may be stabilized by migalastat thereby restoring their trafficking to lysosomes and their intralysosomal activity.
GALAFOLD™ is an alpha-galactosidase A (alpha-Gal A) pharmacological chaperone indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
Metrochem API Expertise:
Metrochem API Pvt Ltd is a leading manufacturer and supplier of Active Pharmaceutical Ingredients (APIs) through our manufacturing sites in Hyderabad and Vizag. Our API facilities have been inspected and approved by several global regulatory bodies including the US FDA, Japan PMDA, KFDA, and meet the cGMP compliance standards. Adapting quickly to the ever-evolving demands, we have emerged as one of the fastest growing organization in the pharmaceutical industry across the India. To cater our customer high volume requirements, we have dedicated each of our 6 manufacturing facilities to our 3 core product groups – APIs, Pelletization& Intermediates – reinforced by a well equipped, centralized Research & Development laboratory.
We have been successfully supplying APIs all over the globe for more than 15 years. Total reactor volume exceeds 1200 KL which includes pilot and multi-purpose units. We are capable of executing extensive API requirements for our customers. Metrochem API Pvt Ltd has an experienced team of highly skilled chemists, experienced regulatory professionals and supply chain experts, who are ready to handle your API manufacturing requirements with expert care. We also offer Contract Manufacturing, Contract Research, and Custom Synthesis services. We have a stand-alone R&D center located in Hyderabad with State-of-the-art analytical facilities and over 90 scientists.We have extensively worked on and delivered new products across a wide variety of therapeutic segments and as a company, we have built strong expertise in new product development.
Disclaimer : Metrochem API Pvt Ltd respects the intellectual property rights of others. Some of the Active Pharmaceuticals Ingredients (APIs) listed in the Metrochem’s product list or brochure or in the product list uploaded in Metrochem’s website may be protected by a valid patent in India or in any other countries concerned. However, mentioning of the name of such product(s) in the product brochure or the product list shall not be construed as infringement in any manner whatsoever. Without prejudice to the foregoing, Metrochem’s supply of API being protected by a valid product patent, if any, is intended solely for uses reasonably relating to the development and submission of information required under any law for the time being inforce, in India, or in a country other than India that regulates the manufacture, use, sale or import of the said product, strictly under the conditions as set forth in Section 107A of the Indian patents Act, 1970, as amended in 2005, or an equivalent provision (Bolar exemption) thereof in a country other than India. The disclaimer is applicable till the expiration of the corresponding product patent(s), if any, in the respective. countries concerned. None of the products will be supplied to the countries wherein this could be in conflict with existing laws. However, the final responsibility lies exclusively with the buyer/ultimate customer.