FDA approves nonsteroidal treatment for Duchenne muscular dystrophy
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- Category: FDA
The U.S. Food and Drug Administration approved Duvyzat (givinostat) oral medication for the treatment of Duchenne Muscular Dystrophy (DMD) in patients six years of age and older. Duvyzat is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. It is a histone deacetylase (HDAC) inhibitor that works by targeting pathogenic processes to reduce inflammation and loss of muscle.
FDA approves first gene therapies to treat patients with sickle cell disease
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- Category: FDA
Today, the U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an innovative advancement in the field of gene therapy.
FDA approves first therapy for rare type of non-cancerous tumors
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- Category: FDA
The U.S. Food and Drug Administration approved Ogsiveo (nirogacestat) tablets for adult patients with progressing desmoid tumors who require systemic treatment. Ogsiveo is the first drug to be approved for the treatment of patients with desmoid tumors, a rare subtype of soft tissue sarcomas.
First electronic product information (ePI) published for selected human medicines
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- Category: EMA
The Heads of Medicines Agencies (HMA), the European Commission (EC) and EMA have published for the first time electronic product information (ePI) for selected human medicines harmonised across the European Union (EU).
The product information of a medicine includes its summary of product characteristics, labelling and package leaflet.
FDA approves first treatment for patients with rare inherited blood clotting disorder
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- Category: FDA
Today, the U.S. Food and Drug Administration approved Adzynma, the first recombinant (genetically engineered) protein product indicated for prophylactic (preventive) or on demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), a rare and life-threatening blood clotting disorder.
FDA takes action on updated mRNA COVID-19 vaccines to better protect against currently circulating variants
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- Category: FDA
Today, the U.S. Food and Drug Administration took action approving and authorizing for emergency use updated COVID-19 vaccines formulated to more closely target currently circulating variants and to provide better protection against serious consequences of COVID-19, including hospitalization and death. Today's actions relate to updated mRNA vaccines for 2023-2024 manufactured by ModernaTX Inc. and Pfizer Inc.
FDA approves first oral treatment for postpartum depression
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- Category: FDA
The U.S. Food and Drug Administration approved Zurzuvae (zuranolone), the first oral medication indicated to treat postpartum depression (PPD) in adults. PPD is a major depressive episode that typically occurs after childbirth but can also begin during the later stages of pregnancy. Until now, treatment for PPD was only available as an IV injection given by a health care provider in certain health care facilities.
More Pharma News ...
- FDA approves first nonprescription daily oral contraceptive
- FDA approves first gene therapy for treatment of certain patients with Duchenne muscular dystrophy
- FDA announces additional steps to modernize clinical trials
- FDA approves first drug to treat agitation symptoms associated with dementia due to Alzheimer's disease
- FDA approves first respiratory syncytial virus (RSV) vaccine
- FDA approves cell therapy for patients with blood cancers to reduce risk of infection following stem cell transplantation
- FDA approves first over-the-counter naloxone nasal spray