FDA
-
News
SICKLE CELL DISEASE | FDA Grants Orphan Drug Designation to AND017
Announced by KIND Pharmaceuticals on October 25, 2024, ODD is granted to a drug or biological product to prevent, diagnose, or treat a rare disease or condition that affects fewer…
Read More » -
News
NEW APPROVAL | FDA Endorses Piasky for Paroxysmal Nocturnal Hemoglobinuria
Piasky, developed by Roche, is a monoclonal antibody that targets complement protein C5. Crovalimab binds to C5 to prevent its cleavage, thus preventing the formation of the membrane attack complex…
Read More » -
News
PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | Ruxoprubart Scores FDA Orphan Drug Designation for PNH Treatment
FDA’s recognition of Ruxoprubart as an orphan drug for PNH underscores its potential to fulfill a crucial need for individuals grappling with this disease condition. Ruxoprubart is currently undergoing a…
Read More » -
Clinical News
SICKLE CELL DISEASE | FDA Approves Two Landmark Gene Therapies for the Treatment of the Disorder
The two therapies are Casgevy (exa-cel) by Vertex Pharmaceuticals and CRISPR Therapeutics – the world’s first drug to utilize the revolutionary CRISPR gene-editing system which earned its creators the 2020…
Read More » -
News
GENE THERAPY | FDA Decision on Lovo-cel for SCD Now Expected by Year’s End
The regulatory agency has agreed to review an application from lovo-cel’s developer, Bluebird Bio, requesting the gene therapy’s approval for SCD patients ages 12 and older. Moreover, the FDA has given the application priority…
Read More » -
News
DIAGNOSTICS | FDA Clears Baebies Disposable Enzyme Deficiency G6PD Test
Using a 50 µL blood sample (one drop of blood), FINDER delivers results approximately 15 minutes after sample introduction. Test results display G6PD enzyme activity in units per gram…
Read More » -
News
ADVANCED THERAPIES | Vertex, CRISPR To Submit Exa-Cel to FDA For β-Thalassaemia And Sickle Cell Disease
Vertex will submit its biologics licensing application (BLA) for exa-cel for rolling review, beginning in November 2022 and expects to complete the submission package by the end of Q1 2023.…
Read More » -
Clinical News
PYRUVATE KINASE DEFICIENCY | FDA Approves First Drug For The Rare Inherited Anemia
The US Food and Drug Administration (FDA) has announced the approval of Mitapivat (PYRUKYND®) tablets as treatment for haemolytic anemia in adults with pyruvate kinase (PK) deficiency. The oral PK…
Read More » -
News
MITAPIVAT | FDA Grants Priority Review To New Drug For Pyruvate Kinase Deficiency
The NDA was granted a Priority Review designation and has been given a Prescription Drug User Fee Act (PDUFA) action date of February 17, 2022, accelerating the review time from…
Read More » -
News
CDC, FDA Authorize Third Dose Of COVID-19 Vaccines For The Immunocompromised In The US
The U.S. Food and Drug Administration (FDA) and the U.S. Centers for Disease Control and Prevention (CDC) have both recommended on August 16 that certain immunocompromised people receive a third…
Read More »