Drugs.com

MARCH 15, 2024

FRIDAY, March 15, 2024 -- Millions of Americans whose livers develop scar tissue due to a common disease now have the first approved drug, Rezdiffra, to treat the condition, the U.S. Food and Drug Administration announced Thursday. The condition is.

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Drugs.com

FEBRUARY 18, 2025

Food and Drug Administration has approved Penmenvy (Meningococcal Groups A, B, C, W, and Y Vaccine) for active immunization against invasive meningococcal disease (IMD), according to a press release from. TUESDAY, Feb. 18, 2025 -- The U.S.

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BioPharma Drive: Drug Pricing

AUGUST 29, 2023

A broadened clearance for Reblozyl in myelodysplastic syndromes should help Bristol Myers offset the looming loss of revenue from top-selling medicines set to soon lose market exclusivity.

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Drugs.com

DECEMBER 8, 2023

Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology. FRIDAY, Dec. 8, 2023 -- The U.S. Casgevy, developed by Vertex.

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Drugs.com

DECEMBER 8, 2023

Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology.Casgevy, developed by Vertex. FRIDAY, Dec. 8, 2023 -- The U.S.

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BioPharma Drive: Drug Pricing

FEBRUARY 24, 2025

Mirum gained the drug, which it will sell as Ctexli for adults with cerebrotendinous xanthomatosis, in a 2023 deal with Travere Therapeutics.

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Drugs.com

JANUARY 16, 2025

Food and Drug Administration has approved Omvoh (mirikizumab-mrkz) for the treatment of moderately to severely active Crohn disease in adults, according to a press release from Eli Lilly and Company.Omvoh targets. THURSDAY, Jan. 16, 2025 -- The U.S.

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Drugs.com

FEBRUARY 18, 2025

Food and Drug Administration has approved Penmenvy (Meningococcal Groups A, B, C, W, and Y Vaccine) for active immunization against invasive meningococcal disease (IMD), according to a press release from. TUESDAY, Feb. 18, 2025 -- The U.S.

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BioPharma Drive: Drug Pricing

AUGUST 29, 2024

Nipocalimab is one of several drugs J&J has forecast for multi-billion dollar sales. The company’s initial application is for its use in myasthenia gravis.

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BioPharma Drive: Drug Pricing

JANUARY 18, 2024

The biotech will receive $500 million upon FDA approval of its rare disease drug acoramidis after selling 5% sales royalties to two investment firms.

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thought leadership

FEBRUARY 26, 2025

Rare diseases are defined in the Orphan Drug Act as diseases or conditions that affect less than 200,000 people in the United States. Evaluating and developing treatments for rare diseases remains a key priority for FDA and is incentivized through the Orphan Drug Act.

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SCIENMAG: Medicine & Health

OCTOBER 31, 2023

— A new drug developed by professors from the School of Pharmacy and Pharmaceutical Sciences at Binghamton University has received Food and Drug Administration (FDA) approval for the treatment of patients with Duchenne muscular dystrophy (DMD), a common genetic disease that mostly affects young boys.

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BioPharma Drive: Drug Pricing

JUNE 21, 2023

Clearance of a subcutaneous form of Argenx’s medicine, known as Vyvgart, could help pad a launch that’s already exceeded Wall Street expectations.

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PLOS: DNA Science

MARCH 28, 2024

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression.

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FDA Law Blog: Drug Discovery

NOVEMBER 10, 2024

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

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SCIENMAG: Medicine & Health

FEBRUARY 9, 2024

CLEVELAND—University Hospitals (UH) Brain Health & Memory Center is now treating patients with LEQEMBI® (lecanemab), a Food and Drug Administration-approved medication for the treatment of Alzheimer’s disease.

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The Pharma Data

AUGUST 9, 2021

Food and Drug Administration approved Nexviazyme (avalglucosidase alfa-ngpt) for intravenous infusion to treat patients 1 year of age and older with late-onset Pompe disease. deputy director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research.

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Chemical Biology and Drug Design

OCTOBER 10, 2023

Abstract Human immunodeficiency virus (HIV) causes acquired immunodeficiency syndrome (AIDS), a lethal disease that is prevalent worldwide. Viral reverse transcriptase (RT) is an excellent target for drug intervention. Nucleoside reverse transcriptase inhibitors (NRTIs) were the first class of approved antiretroviral drugs.

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Drug Target Review

JUNE 21, 2023

Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.

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Covalent Modifiers

APRIL 28, 2023

Food and Drug Administration (FDA) approved small molecules. CysDB is publicly available at [link] and features measures of identification for 62,888 cysteines (24% of the cysteinome), as well as annotations of functionality, druggability, disease relevance, genetic variation, and structural features.

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The Pharma Data

JANUARY 17, 2021

plans to create a global R&D achievement based on innovations of inflammation–fibrosis treatment, Triple-acting new drug for NASH (non-alcoholic steatohepatitis) treatment as well as various other innovations in metabolic disease, oncology and rare disease fields. are expected to be approved by the U.S. .

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The Pharma Data

JANUARY 17, 2021

Food and Drug Administration (FDA) approved Janssen Pharmaceuticals ’ (a Johnson and Johnson company) Darzalex Faspro for adults with newly diagnosed light chain amyloidosis. It was approved in combination with bortezomib, cyclophosphamide and dexamethasone (D-VCd). Michael Vi/Shutterstock.

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Metabolite Tales Blog

JANUARY 26, 2023

Metabolism of 2022 FDA approved small molecule drugs – Part 1 Does CYP3A4 still rule? By Julia Shanu-Wilson It won’t come as much surprise to learn that of the 17 small molecules* approved by the FDA in 2022, CYP3A4 was the major player in drug metabolism.

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Drug Target Review

JANUARY 8, 2024

It is no mystery that as we age our health starts to deteriorate, and we become increasingly susceptible to diseases. Through years of scientific exploration and research, we now know there are several biological changes that make our bodies more susceptible to disease or injury, and we can target those with therapeutic interventions.

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The Pharma Data

AUGUST 7, 2021

Approval is based on positive Phase 3 data demonstrating improvements in key disease burden measures and establishing its safety profile Nexviazyme specifically targets the M6P receptor, the key pathway for enzyme replacement therapy, to effectively clear glycogen build-up in muscle cells.

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The Pharma Data

APRIL 26, 2022

director of the FDA’s Center for Drug Evaluation and Research. Today’s approval of the first COVID-19 therapeutic for this population demonstrates the agency’s commitment to that need.”. Learn more about FDA-approved and authorized COVID-19 vaccines. The FDA granted approval to Gilead Sciences Inc.

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FDA Law Blog: Drug Discovery

MARCH 26, 2024

s Frank Sasinowski , James Valentine and Charles Raver are honored to have aided Italfarmaco in the development and approval of this new drug, and to be part of the effort to expand treatment options for the young men and boys living with Duchenne, and the families of those affected by Duchenne. Hyman, Phelps & McNamara, P.C.’s

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The Pharma Data

AUGUST 19, 2021

Food and Drug Administration (FDA) approval of the VENTANA MMR RxDx Panel, advancing the company’s commitment to personalised healthcare through tests that determine which patients are most likely to benefit from specific and targeted therapies. today announced U.S.

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The Pharma Data

JANUARY 21, 2021

Food and Drug Administration (FDA) gave a greenlight for ViiV Healthcare ’s Cabenuva. Today’s FDA approval of Cabenuva represents a shift in the way HIV is treated, offering people living with HIV a completely new approach to care,” said Lynn Baxter, Head of North America, ViiV Healthcare.

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The Pharma Data

NOVEMBER 29, 2021

Food and Drug Administration today approved Cytalux (pafolacianine), an imaging drug intended to assist surgeons in identifying ovarian cancer lesions. The drug is designed to improve the ability to locate additional ovarian cancerous tissue that is normally difficult to detect during surgery. Related Information.

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ASPET

OCTOBER 19, 2023

Cardiovascular disease, chronic kidney disease, and anemia are known to adversely affect each other. Inflammation is commonly involved in these diseases. Dimethyl fumarate (DMF) is an FDA-approved antioxidant and anti-inflammatory agent. DMF also improved anemia.

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ASPET

JUNE 27, 2024

CB1 and CB2 agonists exhibit broad anti-inflammatory properties, suggesting their potential to treat inflammatory diseases. The primary endpoint was the peak effect (Emax) on a bipolar Drug Liking visual analog scale (VAS). Dose-dependent increases in ratings of Drug Liking were observed with 60 and 120mg lenabasum.

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Codon

NOVEMBER 3, 2024

Physicians working in the early 20th century had little choice but to treat the world’s most rampant infectious disease with methods such as these. But even now, more than a century later, TB remains the deadliest infectious disease on Earth, killing about 1.2 million people every year.

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PLOS: DNA Science

OCTOBER 17, 2024

FDA approved a treatment for type 3 of the ultra-rare genetic disease September 20. The quest has been ongoing for three decades, said Laurie Turner, Family Services Manager of the National Niemann-Pick Disease Foundation. ” Miplyffa is the first drug developed to treat NPD3 specifically. .”

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The Pharma Data

OCTOBER 8, 2021

Food and Drug Administration approved Rethymic for the treatment of pediatric patients with congenital athymia, a rare immune disorder. Rethymic is the first thymus tissue product approved in the U.S. director of the FDA’s Center for Biologics Evaluation and Research. Today, the U.S. federal government.

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The Pharma Data

AUGUST 15, 2021

Food and Drug Administration (FDA) has approved TICOVAC (tick-borne encephalitis (TBE) vaccine) for active immunization to prevent TBE in individuals 1 year of age and older. 1 TICOVAC is the only FDA-approved vaccine to help protect U.S. 1 TICOVAC is the only FDA-approved vaccine to help protect U.S.

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The Pharma Data

NOVEMBER 21, 2021

Food and Drug Administration approved Voxzogo (vosoritide) injection to improve growth in children five years of age and older with achondroplasia and open epiphyses (growth plates), meaning these children still have the potential to grow. The FDA, an agency within the U.S. Today, the U.S.

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