FDA Approval and Medicine - Clinical Research Informer

FDA approves new ALS medicine in precedent-setting decision

Bio Pharma Dive

APRIL 25, 2023

Biogen’s drug failed the key study meant to show it can slow the nerve-destroying disease, but the drug’s effect on a protein of interest led the FDA to conditionally clear it for market.

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US FDA approves Blueprint Medicines’ indolent systemic mastocytosis therapy

Pharmaceutical Technology

MAY 23, 2023

The US Food and Drug Administration (FDA) has granted approval for Blueprint Medicines’ Ayvakit (avapritinib) as the first and only treatment for indolent systemic mastocytosis (ISM) in adult patients.

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FDA approves first microbiota-based treatment

Bio Pharma Dive

DECEMBER 1, 2022

The regulatory OK, a milestone for microbiome-based drug research, is for a medicine from Ferring Pharma that treats a recurrent type of gut infection.

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Symvess: Humacyte’s FDA-Approved Bioengineered Solution for Vascular Trauma

XTalks

JANUARY 7, 2025

With FDA approval granted, Humacyte is working to make Symvess widely available in hospitals, trauma centers and military facilities, offering new treatment options for patients with vascular injuries. Symvess does carry risks, including thrombosis, graft rupture and infection.

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BioMarin finally secures FDA approval of hemophilia gene therapy

Bio Pharma Dive

JUNE 29, 2023

After a prolonged journey, the medicine, known as Roctavian, is now cleared for certain patients with hemophilia A, the more common form of the rare bleeding disorder.

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US FDA approves Reata Pharmaceuticals’ SKYCLARYS for Friedreich’s ataxia

Pharmaceutical Technology

MARCH 2, 2023

The FDA granted Orphan Drug, Fast Track, and Rare Pediatric Disease Designations for the medication. The European Medicines Agency (EMA) is also reviewing Reata’s marketing authorisation application (MAA) for SKYCLARYS in Europe. There are three more drug candidates with major trial readouts that are expected in 2023.

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In historic move, FDA approves a closely watched and controversial Alzheimer's drug

Bio Pharma Dive

JUNE 7, 2021

With the decision, the agency cleared the way for what many predict will become a lucrative and highly sought-after option — though fierce debate continues over whether the medicine, Biogen's aducanumab, actually benefits patients' daily lives.

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AstraZeneca, Avillion get FDA approval for new ‘rescue’ drug for asthma

Bio Pharma Dive

JANUARY 11, 2023

Airsupra joins a growing portfolio of new drugs that are helping the British drugmaker offset wilting sales of older respiratory medicines like Pulmicort.

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US FDA approves GSK’s Jesduvroq to treat anaemia of CKD

Pharmaceutical Technology

FEBRUARY 2, 2023

In June 2020, Japan’s Ministry of Health, Labour and Welfare approved daprodustat tablets to treat patients with anaemia of CKD. The European Medicines Agency is currently reviewing a marketing authorisation application for daprodustat and a regulatory decision is expected in the first half of this year.

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FDA approves IND application for Everest’s cancer vaccine

Pharmaceutical Technology

MARCH 24, 2025

The FDA has granted approval for the IND application of Everest Medicines tumour-associated antigen (TAA) vaccine, EVM14.

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FDA approves Lilly’s obesity med Zepbound to treat obstructive sleep apnoea

Pharmaceutical Technology

DECEMBER 23, 2024

Lillys Zepbound has become the first prescription medicine approved to treat adults with moderate to severe obstructive sleep apnoea (OSA) and obesity.

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Lexicon gets FDA approval for Inpefa to treat heart failure

Pharmaceutical Technology

MAY 29, 2023

Lexicon senior vice president and chief medical officer Dr Craig Granowitz said: “With today’s FDA approval, Inpefa is now a valuable option for physicians to consider when treating patients transitioning out of the hospital and working to break the cycle of repeated hospitalisation.”

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AstraZeneca-Daiichi Sankyo’s Enhertu gets US FDA approval for breast cancer

Pharmaceutical Technology

AUGUST 8, 2022

AstraZeneca Oncology Business Unit executive vice-president Dave Fredrickson said: “The rapid approval of Enhertu in HER2-low metastatic breast cancer by the FDA underscores the urgency to bring this transformational medicine to patients as quickly as possible.

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Seagen, Genmab win speedy FDA approval for cervical cancer drug

Bio Pharma Dive

SEPTEMBER 21, 2021

Tivdak, a type of drug known as an antibody-drug conjugate, is the fourth approved medicine for Seagen and its third to reach market since December 2019.

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Bristol Myers gets key FDA approval for bone marrow disease drug

Bio Pharma Dive

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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Novo keeps price in line with FDA approval of second obesity shot

Bio Pharma Dive

JUNE 7, 2021

Wegovy, a weekly drug, joins daily Saxenda in the pharma's portfolio, which could soon be under pressure from an experimental Lilly medicine.

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FDA approves drugs from Gilead, Roche, TG Therapeutics to close 2022

Bio Pharma Dive

JANUARY 3, 2023

The agency’s main review office cleared 37 new medicines last year, its lowest total since 2016 and well below 2021’s mark of 50.

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After setback, Biohaven to seek approval of neurological disorder drug

Bio Pharma Dive

SEPTEMBER 23, 2024

With fresh, positive data in hand, Biohaven now believes its medicine could be the first to receive FDA approval for spinocerebellar ataxia.

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FDA approves Novartis’ copycat of blockbuster Biogen drug

Bio Pharma Dive

AUGUST 25, 2023

According to one analyst, the approval was the last hurdle keeping Sandoz’s Tyruko from directly competing in the U.S. market against Biogen’s inflammation-regulating medicine Tysabri.

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Catalent Signs Commercial Supply Agreement with Blueprint Medicines Following FDA Approval of GAVRETO™ (pralsetinib)

Pharma Mirror

NOVEMBER 24, 2020

Catalent, the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products, today announced that it had entered into a commercial supply agreement with Blueprint Medicines following FDA approval of GAVRETO™ (pralsetinib).

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After delay, Bluebird submits sickle cell gene therapy for FDA approval

Bio Pharma Dive

APRIL 24, 2023

Submission of the application comes weeks after rivals Vertex and CRISPR filed their gene editing medicine for the disease with the agency.

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FDA Approves Powerful New Drug to Counter Obesity Epidemic

AuroBlog - Aurous Healthcare Clinical Trials blog

NOVEMBER 12, 2023

The US Food and Drug Administration (FDA) on Wednesday announced it had approved Eli Lilly’s highly anticipated weight loss drug Zepbound, the latest entrant in a field of powerful – and lucrative – new obesity medicines.

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Bristol Myers wins FDA approval for first drug to treat inherited heart condition

Bio Pharma Dive

APRIL 28, 2022

Bristol Myers paid $13 billion to acquire the drug's maker, MyoKardia, and expects a top-selling medicine. But a tricky dosing regimen, as well as safety concerns, could challenge the pharmaceutical giant's lofty forecast.

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Merck, Bayer win FDA approval for heart failure drug

Bio Pharma Dive

JANUARY 20, 2021

The drugmakers could face an uphill battle, however, to market the medicine, which showed a more modest benefit than expected in a key study.

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FDA hits Ozempic and Mounjaro counterfeit compounders with warning letters

Pharmaceutical Technology

SEPTEMBER 25, 2024

Some counterfeits contain bacteria and have high impurity levels or different chemical structures to Lilly's FDA-approved medicines.

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Sage, reeling from depression drug decision, to lay off 40% of workforce

Bio Pharma Dive

AUGUST 31, 2023

Two founding executives are leaving Sage alongside a major restructuring announced weeks after the FDA approved the medicine, Zurzuvae, for much narrower use than the company had hoped.

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Akebia tries for FDA approval of anemia drug after mixed data

Bio Pharma Dive

MARCH 30, 2021

That result, and the surprisingly close FDA scrutiny of a rival medicine from FibroGen, makes the approval prospects of both treatments uncertain. The drugmaker's pill fell short on a critical safety measure in two large trials last fall.

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AGEPHA Pharma’s LODOCO gets US FDA approval for cardiovascular disease

Pharmaceutical Technology

JUNE 21, 2023

It can be used as a monotherapy or along with cholesterol-lowering medicines. It is claimed to be the first anti-inflammatory atheroprotective cardiovascular treatment to receive approval from the regulator. It has been designed to target residual inflammation as an underlying cause of ASCVD.

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AstraZeneca and MSD’s Lynparza combo bags FDA approval for prostate cancer

Pharmaceutical Technology

JUNE 1, 2023

Safety and tolerability were in line with that observed in prior trials and the known profiles of the medicines. In comparison, the rPFS and OS for those on placebo with Zytiga and prednisone was 8 months and 23 months, respectively. A prior orchiectomy or receipt of gonadotropin-releasing hormone (GnRH) analogs was also necessary.

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Ctexli (Chenodiol) Gains FDA Approval as First CTX Treatment

XTalks

FEBRUARY 25, 2025

Mirum Pharmaceuticals, a biotech innovator known for developing therapies for rare metabolic disorders, now has FDA approval for its new treatment, Ctexli (chenodiol) tablets the first and only medication approved for cerebrotendinous xanthomatosis (CTX) in adults.

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Ryoncil, the first FDA-approved mesenchymal stromal cell therapy

Pharmaceutical Technology

MARCH 3, 2025

Regenerative medicine, a rapidly advancing field, can be generally defined as a therapeutic methodology for the repair, regeneration, or replacement of damaged or diseased human cells and tissues.

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Pfizer’s sNDA for Cibinqo receives FDA approval for atopic dermatitis

Pharmaceutical Technology

FEBRUARY 13, 2023

Pfizer has received the US Food and Drug Administration (FDA) approval for its supplemental New Drug Application (sNDA) for Cibinqo (abrocitinib) to treat a type of atopic dermatitis (AD). The post Pfizer’s sNDA for Cibinqo receives FDA approval for atopic dermatitis appeared first on Pharmaceutical Technology.

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Alnylam wins FDA approval of rare disease drug in step toward profitability

Bio Pharma Dive

JUNE 14, 2022

Amvuttra, a transthyretin amyloidosis medicine previously known as vutrisiran, is viewed by biotech analysts as important to the company’s goal of ending years of financial losses.

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US FDA approves Vertex’s Orkambi for cystic fibrosis in children

Pharmaceutical Technology

SEPTEMBER 5, 2022

Earlier, the therapy had obtained FDA approval for usage in CF patients aged two years and above with two F508del mutation copies. The latest approval for use in children aged 12 to below 24 months is based on an open-label, multicentre Phase III clinical trial for 24 weeks.

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Coherus wins FDA approval for second Lucentis biosimilar, its third drug

Bio Pharma Dive

AUGUST 3, 2022

The biotech company secured a valuable interchangeable designation for the copycat medicine, which it plans to launch later this year.

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FDA approves drugs from Vertex, Novo, Bristol Myers to wrap 2024

Bio Pharma Dive

JANUARY 2, 2025

The agency’s main drug review office cleared 50 novel medicines last year, short of 2018’s record total but on the higher end of recent annual tallies.

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Teva, MedinCell receive FDA approval for Uzedy to treat schizophrenia

Pharmaceutical Technology

MAY 1, 2023

“The approval of Uzedy is a culmination of a multidisciplinary effort across Teva and MedinCell to bring this important treatment to market. The regulatory approval is based on the data obtained from the Risperidone Subcutaneous Extended-Release Study (RISE) and the SHINE Phase III trials.

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FDA approves GSK’s new bone cancer drug, helping validate a billion-dollar bet

Bio Pharma Dive

SEPTEMBER 18, 2023

The drug, known scientifically as momelotinib, is now cleared to treat myelofibrosis and will compete against rival medicines sold by Incyte and Bristol Myers Squibb.

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FDA approves Ipsen’s Bylvay for cholestatic pruritus due to ALGS

Pharmaceutical Technology

JUNE 14, 2023

Ipsen has received US Food and Drug Administration (FDA) approval for Bylvay (odevixibat) to treat patients aged 12 months and above with cholestatic pruritus caused by Alagille syndrome (ALGS). Ipsen is also seeking marketing approval from the European Medicines Agency, with a final decision expected in the second half of 2023.

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US FDA approves Pharming’s Joenja to treat APDS

Pharmaceutical Technology

MARCH 27, 2023

It is claimed to be the first and only therapy to receive approval in the US for the treatment of APDS, a rare and progressive primary immunodeficiency. Leniolisib’s marketing authorisation application (MAA) is currently under review with the Committee for Human Medicinal Products (CHMP) of the European Medicines Agency (EMA).

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Vertex, CRISPR score landmark FDA approval for sickle cell disease gene therapy Casgevy

Fierce Pharma

DECEMBER 8, 2023

| Vertex Pharmaceuticals and CRISPR Therapeutics have won an historic FDA approval for their sickle cell disease gene therapy Casgevy. The transformational treatment is a potential cure for the debilitating and life-threatening disease which affects more than 100,000 in the United States, most of them Black.

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Pfizer heralds “paradigm shift” in hemophilia B gene therapy with FDA approval

BioPharma Reporter

APRIL 30, 2024

million gene therapy for hemophilia B just got FDA approval. Hereâs what it means for patients and the future of medicine Pfizer's groundbreaking $3.5

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Blueprint Medicines announces financing partnerships for $1.25bn

Pharmaceutical Technology

JULY 1, 2022

Blueprint Medicines has entered strategic financing partnerships for up to $1.25bn with life sciences-focused investors Sixth Street and Royalty Pharma. Ayvakit has received the US Food and Drug Administration (FDA) approval to treat advanced systemic mastocytosis in adults. The collaboration with Sixth Street has three parts.

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Blue Water Biotech shores up portfolio with purchase of six FDA-approved treatments

Pharmaceutical Technology

JUNE 14, 2023

All six treatments are US Food and Drug Administration (FDA) approved. The six newly acquired medicines join Entadfi (finasteride and tadalafil) in Blue Water’s portfolio. Blue Water bought the FDA-approved benign prostatic hyperplasia treatment from Veru for a total cost of $100m in April 2023. “We

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FDA approves new ALS medicine in precedent-setting decision

US FDA approves Blueprint Medicines’ indolent systemic mastocytosis therapy

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FDA approves first microbiota-based treatment

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Symvess: Humacyte’s FDA-Approved Bioengineered Solution for Vascular Trauma

BioMarin finally secures FDA approval of hemophilia gene therapy

US FDA approves Reata Pharmaceuticals’ SKYCLARYS for Friedreich’s ataxia

In historic move, FDA approves a closely watched and controversial Alzheimer's drug

AstraZeneca, Avillion get FDA approval for new ‘rescue’ drug for asthma

US FDA approves GSK’s Jesduvroq to treat anaemia of CKD

FDA approves IND application for Everest’s cancer vaccine

FDA approves Lilly’s obesity med Zepbound to treat obstructive sleep apnoea

Lexicon gets FDA approval for Inpefa to treat heart failure

AstraZeneca-Daiichi Sankyo’s Enhertu gets US FDA approval for breast cancer

Seagen, Genmab win speedy FDA approval for cervical cancer drug

Bristol Myers gets key FDA approval for bone marrow disease drug

Novo keeps price in line with FDA approval of second obesity shot

FDA approves drugs from Gilead, Roche, TG Therapeutics to close 2022

After setback, Biohaven to seek approval of neurological disorder drug

FDA approves Novartis’ copycat of blockbuster Biogen drug

Catalent Signs Commercial Supply Agreement with Blueprint Medicines Following FDA Approval of GAVRETO™ (pralsetinib)

After delay, Bluebird submits sickle cell gene therapy for FDA approval

FDA Approves Powerful New Drug to Counter Obesity Epidemic

Bristol Myers wins FDA approval for first drug to treat inherited heart condition

Merck, Bayer win FDA approval for heart failure drug

FDA hits Ozempic and Mounjaro counterfeit compounders with warning letters

Sage, reeling from depression drug decision, to lay off 40% of workforce

Akebia tries for FDA approval of anemia drug after mixed data

AGEPHA Pharma’s LODOCO gets US FDA approval for cardiovascular disease

AstraZeneca and MSD’s Lynparza combo bags FDA approval for prostate cancer

Ctexli (Chenodiol) Gains FDA Approval as First CTX Treatment

Ryoncil, the first FDA-approved mesenchymal stromal cell therapy

Pfizer’s sNDA for Cibinqo receives FDA approval for atopic dermatitis

Alnylam wins FDA approval of rare disease drug in step toward profitability

US FDA approves Vertex’s Orkambi for cystic fibrosis in children

Coherus wins FDA approval for second Lucentis biosimilar, its third drug

FDA approves drugs from Vertex, Novo, Bristol Myers to wrap 2024

Teva, MedinCell receive FDA approval for Uzedy to treat schizophrenia

FDA approves GSK’s new bone cancer drug, helping validate a billion-dollar bet

FDA approves Ipsen’s Bylvay for cholestatic pruritus due to ALGS

US FDA approves Pharming’s Joenja to treat APDS

Vertex, CRISPR score landmark FDA approval for sickle cell disease gene therapy Casgevy

Pfizer heralds “paradigm shift” in hemophilia B gene therapy with FDA approval

Blueprint Medicines announces financing partnerships for $1.25bn

Blue Water Biotech shores up portfolio with purchase of six FDA-approved treatments

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