FDA and Treatment - Drug Discovery Digest

FDA panel votes against MDMA therapy for PTSD, citing muddy data and safety concerns

BioPharma Drive: Drug Pricing

JUNE 4, 2024

A group of independent experts wasn't convinced by clinical trial data from company Lykos Therapeutics, which is seeking FDA approval of MDMA-assisted treatment for post-traumatic stress disorder.

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CRISPR therapy for sickle cell approved by FDA in gene editing milestone

BioPharma Drive: Drug Pricing

DECEMBER 8, 2023

In addition to clearing Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy, the FDA also granted an early OK to Bluebird bio’s sickle cell treatment Lyfgenia.

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Vertex, CRISPR therapy for sickle cell passes FDA panel test

BioPharma Drive: Drug Pricing

OCTOBER 31, 2023

The high-profile meeting focused on the theoretical risks of CRISPR gene editing, as both the FDA and its advisory committee appeared convinced by the efficacy of the companies’ exa-cel treatment.

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FDA Approves Susvimo for Diabetic Macular Edema

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FEBRUARY 5, 2025

Food and Drug Administration has approved Susvimo (ranibizumab) 100 mg/mL for treating diabetic macular edema.Susvimo is the first and only FDA-approved treatment shown to maintain vision in people with diabetic. WEDNESDAY, Feb. 5, 2025 -- The U.S.

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Verve gets FDA green light to run base editing study in US

BioPharma Drive: Drug Pricing

OCTOBER 23, 2023

since late last year as the FDA sought more details on Verve’s in vivo treatment for heart disease. The trial, which is ongoing in the U.K. and New Zealand, has been on hold in the U.S.

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FDA Approves Symbravo for Acute Treatment of Migraine in Adults

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FEBRUARY 5, 2025

Food and Drug Administration has approved Symbravo (meloxicam and rizatriptan) for the acute treatment of migraine with or without aura in adults. WEDNESDAY, Feb. 5, 2025 -- The U.S. The approval was based on the results of three phase 3 trials of.

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Galderma wins FDA approval for skin condition treatment

BioPharma Drive: Drug Pricing

AUGUST 13, 2024

Nemluvio is now cleared for adults with the chronic itching condition prurigo nodularis, making it a competitor to Sanofi and Regeneron’s Dupixent.

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FDA Panel Says No to MDMA as Treatment for PTSD

Drugs.com

JUNE 5, 2024

Food and Drug Administration advisory panel on Tuesday voted against recommending the psychedelic MDMA for the treatment of post-traumatic stress disorder (PTSD).In WEDNESDAY, June 5, 2024 -- A U.S. In a 10-1 vote, the panel determined the evidence.

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FDA staff hold ‘major concerns’ with Brainstorm ALS therapy, documents show

BioPharma Drive: Drug Pricing

SEPTEMBER 25, 2023

Agency scientists flagged numerous issues with Brainstorm’s stem cell treatment ahead of a high-profile Wednesday meeting of expert FDA advisers.

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FDA Approves Spravato Nasal Spray for Treatment-Resistant Depression

Drugs.com

JANUARY 22, 2025

WEDNESDAY, Jan. 22, 2025 -- The U.S. Food and Drug Administration has approved Spravato (esketamine) CIII nasal spray for adults living with major depressive disorder who have had an inadequate response to at least two oral antidepressants.

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FDA Panel Says No to Experimental ALS Drug, NurOwn

Drugs.com

SEPTEMBER 28, 2023

Food and Drug Administration on Wednesday voted resoundingly against recommending, NurOwn, a stem cell-based experimental treatment for ALS. Although the FDA isn't bound by the votes of. THURSDAY, Sept. 28, 2023 – An advisory panel to the U.S.

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FDA Approves First Treatment for Severe Frostbite, Aurlumyn (iloprost)

Drugs.com

FEBRUARY 15, 2024

Food and Drug Administration (FDA) on Wednesday approved the first treatment for severe frostbite. THURSDAY, Feb. 15, 2024 -- The U.S. Known as Aurlumyn (iloprost), the injected medication lowers the risk of finger or toe amputation in cases of.

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FDA Wants More Data on Neffy, First Needle-Free Antidote for Severe Allergic Reactions

Drugs.com

SEPTEMBER 20, 2023

Food and Drug Administration (FDA) has opted not to approve a needle-free alternative to the EpiPen for emergency treatment of severe allergic reactions. WEDNESDAY, Sept. 20, 2023 -- In a surprising move, the U.S. Approval of the Neffy nasal spray.

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Nestle gives up on peanut allergy treatment after sluggish sales

BioPharma Drive: Drug Pricing

SEPTEMBER 5, 2023

The FDA-approved therapy, called Palforzia, was little used, leading Nestle to abandon a business it had secured in a $2.6 billion deal three years ago.

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FDA Panel Says No to Experimental ALS Drug

Drugs.com

SEPTEMBER 28, 2023

Food and Drug Administration on Wednesday voted resoundingly against recommending a stem cell-based experimental treatment for ALS. Although the FDA isn't bound by the votes of its advisory. THURSDAY, Sept. 28, 2023 – An advisory panel to the U.S.

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After ‘sobering’ FDA panel, psychedelics supporters wonder what’s next

BioPharma Drive: Drug Pricing

JUNE 7, 2024

A vote against MDMA-assisted therapy set back a high-profile treatment. Proponents argue the panel offered few solutions to longstanding problems, making the path forward unclear.

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FDA Asked to Consider Party Drug MDMA as Treatment for PTSD

Drugs.com

DECEMBER 13, 2023

Food and Drug Administration to approve MDMA, the active ingredient in party drugs like molly and ecstasy, as a treatment for post-traumatic stress disorder (PTSD).When WEDNESDAY, Dec. 13, 2023 -- A California company has asked the U.S. When announcing.

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FDA declines to review Biohaven’s latest drug in setback to pipeline

BioPharma Drive: Drug Pricing

JULY 27, 2023

While the FDA doesn’t think the brain disease treatment merits an evaluation, Biohaven is still pushing for one and has requested a further meeting.

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FDA Approves Dupixent for Chronic Spontaneous Urticaria

Drugs.com

APRIL 23, 2025

Food and Drug Administration has approved Dupixent (dupilumab) for the treatment of adults and adolescents with chronic spontaneous urticaria.The approval is for patients 12 years and older who remain. WEDNESDAY, April 23, 2025 -- The U.S.

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When is a Confirmatory Trial “Underway” or Conducted with “Due Diligence” Enough for Accelerated Approval? FDA Explains Its New Authorities

FDA Law Blog: Drug Discovery

JANUARY 30, 2025

FDAs withdrawal authority when a confirmatory trial is not conducted with due diligence was expanded to include that FDA could specify the conditions for a postapproval study. Considering FDAs new authority to specify the conditions for such confirmatory trials (e.g.,

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FDA Expands Use of Breast Cancer Drug Kisqali

Drugs.com

SEPTEMBER 18, 2024

Food and Drug Administration's expanded approval of the treatment, drug maker Novartis. WEDNESDAY, Sept. 18, 2024 -- Women with early stage breast cancer may now take Kisquali, a medication already approved for advanced disease, following the U.S.

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FDA sets decision dates for Vertex, CRISPR gene editing drug

BioPharma Drive: Drug Pricing

JUNE 9, 2023

The regulator will issue separate verdicts on the pioneering treatment’s use in sickle cell disease and beta thalassemia, with the first expected late this year.

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FDA Says No to MDMA as Therapy for PTSD

Drugs.com

AUGUST 12, 2024

Food and Drug Administration declined to approve it as treatment for. MONDAY, Aug. 12, 2024 -- Advocates for the use of the psychedelic drug MDMA (midomafetamine) to treat mental health conditions were delivered a setback on Friday after the U.S.

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5 FDA decisions to watch in the first quarter

BioPharma Drive: Drug Pricing

JANUARY 4, 2024

The agency could soon clear important new drugs from Eli Lilly and Merck, as well as the first treatment for a common liver disease.

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Ipsen drug for rare liver disease approved by FDA

BioPharma Drive: Drug Pricing

JUNE 11, 2024

The medicine will join Intercept’s Ocaliva as a treatment option for primary biliary cholangitis. Another drug, from the now Gilead-owned CymaBay, could be cleared for the condition by August.

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FDA investigating cancer risk linked to CAR-T cell therapy

BioPharma Drive: Drug Pricing

NOVEMBER 28, 2023

The agency said the benefit of approved treatments like Gilead’s Yescarta still outweighs any such risk, but the alert could slow drugmaker efforts to develop the treatments for wider use.

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Regulator and Funder? FDA’s Orphan Products Grants Program awards significant funding to help move promising treatments through clinical development

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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Novo’s once-weekly insulin rejected by FDA

BioPharma Drive: Drug Pricing

JULY 11, 2024

The FDA’s decision to turn back the drugmaker’s new insulin product narrows Novo’s lead in the U.S. over Eli Lilly, which has a similar diabetes treatment in late-stage testing.

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FDA Warns of Rare Secondary Cancer Risk With CAR-T Therapies

Drugs.com

JANUARY 24, 2024

Food and Drug Administration has told drugmakers to add a boxed warning to a type of cancer treatment called CAR-T therapy, saying the treatment itself may sometimes cause a secondary. WEDNESDAY, Jan. 24, 2024 (Healthday News) -- The U.S.

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FDA Approves TNKase for Acute Ischemic Stroke

Drugs.com

MARCH 6, 2025

Food and Drug Administration has approved TNKase (tenecteplase), a thrombolytic or clot-dissolving agent, for the treatment of acute ischemic stroke in adults.TNKase is delivered as a single five-second. THURSDAY, March 6, 2025 -- The U.S.

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Makary confirmed by Senate as FDA commissioner

BioPharma Drive: Drug Pricing

MARCH 26, 2025

The Johns Hopkins surgeon will helm the agency as layoffs loom and important decisions on treatments for rare and infectious diseases await.

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FDA Approves Landmark Sickle Cell Gene Therapies, Casgevy and Lyfgenia

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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FDA Approves First Treatment for Severe Frostbite

Drugs.com

FEBRUARY 15, 2024

Food and Drug Administration (FDA) on Wednesday approved the first treatment for severe frostbite.Known as Aurlumyn (iloprost), the injected medication lowers the risk of finger or toe amputation in cases of. THURSDAY, Feb. 15, 2024 -- The U.S.

FDA Approval

FDA Approval FDA Treatment Drugs

Madrigal, FDA approval in hand, outlines plan to sell MASH drug

BioPharma Drive: Drug Pricing

MARCH 15, 2024

The company expects initial uptake of Rezdiffra, which costs $47,400 per year, to be slow as doctors and insurers establish the protocols needed to find patients eligible for treatment.

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FDA Approves Linzess to Treat Constipation in Children, Teens

Drugs.com

JUNE 14, 2023

Food and Drug Administration approved Linzess (linaclotide) as a once-daily treatment for pediatric patients ages 6 to 17 years with functional constipation. WEDNESDAY, June 14, 2023 -- The U.S. The approval was based on data from 328 participants.

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FDA Approval FDA Treatment Drugs

FDA Approves Wearable Infusion Device for Advanced Parkinson Disease

Drugs.com

FEBRUARY 6, 2025

Food and Drug Administration has approved Onapgo (apomorphine hydrochloride) injection as the first and only subcutaneous apomorphine infusion device for the treatment of motor fluctuations in patients with. THURSDAY, Feb. 6, 2025 -- The U.S.

FDA Approval

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FDA Approves Blujepa for Uncomplicated Urinary Tract Infections

Drugs.com

MARCH 25, 2025

Food and Drug Administration has approved Blujepa (gepotidacin) for the treatment of uncomplicated urinary tract infections (uUTIs) in adult women (weighing 40 kg or more) and children (12 years and older. TUESDAY, March 25, 2025 -- The U.S.

FDA Approval

FDA Approval FDA Treatment Drugs

BrainStorm’s ALS therapy not effective, FDA panel finds

BioPharma Drive: Drug Pricing

SEPTEMBER 27, 2023

Expert advisers to the agency voted 17-1 that Brainstorm's clinical trial data did not show the company's stem cell treatment was effective for treating ALS.

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Daiichi Sankyo, with new data, to seek FDA approval of lung cancer drug

BioPharma Drive: Drug Pricing

SEPTEMBER 11, 2023

The HER3-targeting treatment could become the next antibody-drug conjugate to emerge from Daiichi Sankyo’s laboratories, after the AstraZeneca-partnered Enhertu.

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FDA broadens use of Novartis’ radiopharma drug Pluvicto

BioPharma Drive: Drug Pricing

MARCH 28, 2025

Pluvicto can now be used before chemotherapy to treat a kind of metastatic prostate cancer, tripling the number of patients who are eligible for treatment.

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BrainStorm’s ALS therapy not effective, FDA panel finds

BioPharma Drive: Drug Pricing

SEPTEMBER 27, 2023

Expert advisers to the agency voted 17-1 that Brainstorm's clinical trial data did not show the company's stem cell treatment was effective for treating ALS.

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FDA Approves First Gene Therapy, Elevidys, to Treat Duchenne Muscular Dystrophy

Drugs.com

JUNE 23, 2023

Food and Drug Administration on Thursday approved the drug Elevidys, the first gene therapy for the treatment of children with Duchenne muscular dystrophy (DMD). The groundbreaking treatment will not be cheap: FRIDAY, June 23, 2023 -- The U.S.

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Roche wins FDA OK for targeted drug in early lung cancer

BioPharma Drive: Drug Pricing

APRIL 19, 2024

The new label for Alecensa makes it the first targeted treatment available after surgery for people with ALK-positive lung tumors, who are at high risk for brain metastases.

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FDA to Import Syphilis Drug, Extencilline, From France Amid Shortage

Drugs.com

JANUARY 11, 2024

11, 2024 -- Amid an ongoing shortage of the first-line treatment for syphilis in the United States, the U.S. THURSDAY, Jan. Food and Drug Administration will allow the importation of a different syphilis drug from a French drugmaker.

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FDA panel votes against MDMA therapy for PTSD, citing muddy data and safety concerns

CRISPR therapy for sickle cell approved by FDA in gene editing milestone

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Vertex, CRISPR therapy for sickle cell passes FDA panel test

Webinars

FDA Approves Susvimo for Diabetic Macular Edema

Verve gets FDA green light to run base editing study in US

FDA Approves Symbravo for Acute Treatment of Migraine in Adults

Galderma wins FDA approval for skin condition treatment

FDA Panel Says No to MDMA as Treatment for PTSD

FDA staff hold ‘major concerns’ with Brainstorm ALS therapy, documents show

FDA Approves Spravato Nasal Spray for Treatment-Resistant Depression

FDA Panel Says No to Experimental ALS Drug, NurOwn

FDA Approves First Treatment for Severe Frostbite, Aurlumyn (iloprost)

FDA Wants More Data on Neffy, First Needle-Free Antidote for Severe Allergic Reactions

Nestle gives up on peanut allergy treatment after sluggish sales

FDA Panel Says No to Experimental ALS Drug

After ‘sobering’ FDA panel, psychedelics supporters wonder what’s next

FDA Asked to Consider Party Drug MDMA as Treatment for PTSD

FDA declines to review Biohaven’s latest drug in setback to pipeline

FDA Approves Dupixent for Chronic Spontaneous Urticaria

When is a Confirmatory Trial “Underway” or Conducted with “Due Diligence” Enough for Accelerated Approval? FDA Explains Its New Authorities

FDA Expands Use of Breast Cancer Drug Kisqali

FDA sets decision dates for Vertex, CRISPR gene editing drug

FDA Says No to MDMA as Therapy for PTSD

5 FDA decisions to watch in the first quarter

Ipsen drug for rare liver disease approved by FDA

FDA investigating cancer risk linked to CAR-T cell therapy

Regulator and Funder? FDA’s Orphan Products Grants Program awards significant funding to help move promising treatments through clinical development

Novo’s once-weekly insulin rejected by FDA

FDA Warns of Rare Secondary Cancer Risk With CAR-T Therapies

FDA Approves TNKase for Acute Ischemic Stroke

Makary confirmed by Senate as FDA commissioner

FDA Approves Landmark Sickle Cell Gene Therapies, Casgevy and Lyfgenia

FDA Approves First Treatment for Severe Frostbite

Madrigal, FDA approval in hand, outlines plan to sell MASH drug

FDA Approves Linzess to Treat Constipation in Children, Teens

FDA Approves Wearable Infusion Device for Advanced Parkinson Disease

FDA Approves Blujepa for Uncomplicated Urinary Tract Infections

BrainStorm’s ALS therapy not effective, FDA panel finds

Daiichi Sankyo, with new data, to seek FDA approval of lung cancer drug

FDA broadens use of Novartis’ radiopharma drug Pluvicto

BrainStorm’s ALS therapy not effective, FDA panel finds

FDA Approves First Gene Therapy, Elevidys, to Treat Duchenne Muscular Dystrophy

Roche wins FDA OK for targeted drug in early lung cancer

FDA to Import Syphilis Drug, Extencilline, From France Amid Shortage

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