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Luspatercept, sold under the brand name Reblozyl, is a medication used for the treatment of anemia in beta thalassemia and myelodysplastic syndromes. [ 5 ] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication .
Luspatercept is a TGFβ ligand that acts to decrease SMAD2 and SMAD3 signaling involved in erythropoeisis and may be used in MDS with anemia that is not responsive to erythrocyte stimulating agents or mild MDS with ring sideroblasts. Luspatercept was shown to decrease the need for transfusions and this effect lasted for a median of 30.6 weeks.
Acceleron Pharma, Inc. is an American clinical stage biopharmaceutical company based in Cambridge, Massachusetts with a broad focus on developing medicines that regulate the transforming growth factor beta (TGF-β) superfamily of proteins, which play fundamental roles in the growth and repair of cells and tissues such as red blood cells, muscle, bone, and blood vessels.
This list of over 500 monoclonal antibodies includes approved and investigational drugs as well as drugs that have been withdrawn from market; consequently, the column Use does not necessarily indicate clinical usage. See the list of FDA-approved therapeutic monoclonal antibodies in the monoclonal antibody therapy page.
The PDUFA date serves as a good first approximation of when a final decision on drug approval can be expected. Sponsors frequently publish PDUFA dates for their pending applications, [1] and while there is no official list of PDUFA dates, [10] several websites collect PDUFA dates from press announcements in a calendar form.
In most countries, a marketing authorisation is valid for a period of 5 years. After this period, one should apply for renewal of the marketing authorisation, usually by providing minimal data proving that quality, efficacy and safety characteristics are maintained and the risk-benefit ratio of the medicinal product is still favourable.
If the Form 356h is missing information, the FDA will reply within 74 days. [3] A BLA asserts that the product is "safe, pure, and potent", the manufacturing facilities are inspectable, and each package of the product bears the license number. Statutory standards for BLA approval are largely the same as those for New Drug Application approval
Non-approval letters were rejections of a drug's application. [2] Approvable and non-approvable letters were covered under Title 21 of the Code of Federal Regulations, section 314.110. [3] In 2018, the FDA replaced approvable letters with Complete Response Letters (CRL) to notify applicants when additional information is required before ...