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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BioPharma Drive: Drug Pricing

OCTOBER 23, 2023

The clearance of the pentavalent shot Penbraya adds to Pfizer’s infectious disease portfolio as it adjusts to slumping COVID-19 vaccine sales.

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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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Drugs.com

JULY 2, 2024

TUESDAY, July 2, 2024 -- A new drug to treat Alzheimer's disease was approved by the U.S. Food and Drug Administration on Tuesday.In clinical trials, donanemab (Kisunla) modestly slowed the pace of thinking declines among patients in the early.

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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

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

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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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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BioPharma Drive: Drug Pricing

MARCH 19, 2024

clearance of Lenmeldy, for a rare and inherited metabolic disease, triggers an additional payout related to Kyowa Kirin’s recent deal to acquire the once high-flying gene therapy developer.

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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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BioPharma Drive: Drug Pricing

FEBRUARY 18, 2025

Penmenvy, which protects against five common types of disease-causing bacteria, is cleared for use in people aged 10 through 25 years old.

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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

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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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

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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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. ITF Therapeutics provides the new drug in the US. Hyperbole or Hope?

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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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Drug Target Review

MARCH 12, 2024

The study’s findings offer new insights into the biochemical language microbes use to influence distant organ systems, which could revolutionise the way researchers approach disease. For example, bile acid treatments have been effective against SARS-Cov-2 infections. This study was published in Cell Reports. Gut Microbes.

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New Drug Approvals

APRIL 11, 2025

2] Fitusiran was approved for medical use in the United States in March 2025. [2] 1] [2] Adverse effects The US Food and Drug Administration prescription label for fitusiran contains a boxed warning for thrombotic events (blood clotting) and gallbladder disease (with some recipients requiring gallbladder removal). [2] 26 March 2025.

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Broad Institute

MAY 16, 2024

But FDA-approved forms of the most commonly used vehicle for packaging and delivering these therapies to target cells, adeno-associated viruses (AAVs), aren’t able to efficiently cross the blood-brain barrier at high levels and deliver therapeutic cargo.

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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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PLOS: DNA Science

JUNE 1, 2023

The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn’t a one-and-done. Earlier results were published in Nature Medicine.

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The Pharma Data

JUNE 2, 2023

Food and Drug Administration (FDA) for the treatment of adult patients with deleterious or suspected deleterious BRCA -mutated ( BRCA m) metastatic castration-resistant prostate cancer (mCRPC). Patients should be selected for therapy based on an FDA-approved companion diagnostic for LYNPARZA. In the U.S., In the U.S.,

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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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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. Later, a new type of non-nucleoside reverse transcriptase inhibitors (NNRTIs) were approved as anti-HIV drugs. Molecular insights of HIV Reverse transcriptase and it's inhibitors.

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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

AUGUST 27, 2020

Cancer is a disease of the genome, driven by genetic mutations within a tumour’s DNA. The convenience of testing a blood sample may also enable more rapid treatment decisions, so that patients can feel reassured they are not losing time to fight their disease.”.

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The Pharma Data

JANUARY 21, 2021

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. At the same time, the FDA approved ViiV’s New Drug Application (NDA) for Vocabria (cabotegravir) 30 mg oral tablets.

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The Pharma Data

SEPTEMBER 7, 2020

These benefits were seen in patients regardless of RET fusion partner or central nervous system involvement.

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The Pharma Data

AUGUST 10, 2020

Genentech’s Evrysdi (risdiplam) has secured FDA approval for the treatment of spinal muscular atrophy (SMA), making it the first oral therapy available for the rare hereditary genetic condition in US and the second overall to be approved for the disease. months at launch.

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The Pharma Data

NOVEMBER 27, 2020

FDA Approves Danyelza (naxitamab-gqgk) for the Treatment of Neuroblastoma. This indication is approved under accelerated approval regulation based on overall response rate and duration of response. This indication is approved under accelerated approval based on overall response rate and duration of response.

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The Pharma Data

AUGUST 21, 2020

The FDA has authorised Novartis’ Kesimpta (ofatumumab) for treating patients with relapsing forms of multiple sclerosis (MS), it has been revealed. . The injectable treatment also covers other forms of MS including clinically isolated syndrome and active secondary progressive disease. It can also be self-administered once a month.

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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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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. References Iversen et al., Curr Drug Metab. 20(4): 254-265.

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The Pharma Data

NOVEMBER 27, 2020

FDA Approves Imcivree (setmelanotide) for Chronic Weight Management in Patients with Obesity Due to POMC, PCSK1 or LEPR Deficiency. Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, announced today that the U.S. BOSTON, Nov.

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