FDA and Therapies - Drug Discovery Digest

Duchenne approval exposes FDA rift over Sarepta gene therapy

BioPharma Drive: Drug Pricing

JUNE 21, 2024

Peter Marks’ decision to override the objections of agency staff and broaden use of Elevidys could have a “lasting impact” on gene therapy as well as the FDA, one analyst wrote.

Therapies

Therapies FDA

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.

FDA

FDA FDA Approval Clinical Trials Therapies

First-of-its-kind cell therapy approved by FDA for rare soft tissue cancer

BioPharma Drive: Drug Pricing

AUGUST 2, 2024

Adaptimmune’s Tecelra is the first TCR cell therapy to reach market and, at $727,000, is also the priciest cellular medicine for cancer in the U.S.

Therapies

Therapies FDA Marketing

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.

FDA

FDA Therapies Treatment

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.

FDA

FDA Therapies Pharmaceuticals Treatment

FDA orders new cancer warnings for CAR-T therapies

BioPharma Drive: Drug Pricing

JANUARY 23, 2024

A black box warning for secondary T cell malignancies should be added to the labeling of all six approved CAR-T therapies, the agency said.

Therapies

Therapies FDA

FDA Advisors Say New Gene Therapy for Sickle Cell Disease, Exa-cel, is Safe

Drugs.com

NOVEMBER 2, 2023

1, 2023 -- A new gene therapy for sickle cell disease was deemed safe by a U.S. The FDA had already decided that the. WEDNESDAY, Nov. Food and Drug Administration advisory panel on Tuesday, paving the way for full approval by early December.

Therapies

Therapies FDA Disease Drugs

Taysha drops lead gene therapy following FDA feedback

BioPharma Drive: Drug Pricing

SEPTEMBER 20, 2023

Additional studies proposed by the FDA would be too challenging, according to Taysha, which simultaneously announced that its partner Astellas has declined to pick up an option for the therapy, called TSHA-120.

Therapies

Therapies FDA

Bristol Myers CAR-T therapy approved by FDA for earlier myeloma use

BioPharma Drive: Drug Pricing

APRIL 5, 2024

The FDA’s clearance comes three weeks after a panel of advisers endorsed expanded use of Abecma despite safety concerns raised by the agency.

FDA

FDA Therapies

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.

FDA Approval

FDA Approval Therapies FDA Treatment

FDA Approves Landmark Sickle Cell Gene Therapies

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.

FDA Approval

FDA Approval Therapies FDA Treatment

With Duchenne decision ahead, FDA’s Marks pushes for speedy gene therapy approvals

BioPharma Drive: Drug Pricing

MAY 24, 2024

But the head of the FDA’s CBER office didn’t tip where the agency stands on potentially broadening use of Sarepta’s Duchenne gene therapy Elevidys.

Therapies

Therapies FDA

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.

Therapies

Therapies FDA Treatment Drugs

5 FDA decisions to watch in the second quarter

BioPharma Drive: Drug Pricing

APRIL 1, 2024

The FDA could soon expand use of two multiple myeloma cell therapies as well as a top-selling medicine from Sanofi and Regeneron, while Pfizer’s years of gene therapy work might finally pay dividends.

FDA

FDA Therapies

Rocket gene therapy rejected by FDA over manufacturing

BioPharma Drive: Drug Pricing

JUNE 28, 2024

The complete response letter follows other recent manufacturing-related rejections, including one for a cell therapy from Abeona Therapeutics.

Therapies

Therapies FDA

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.

FDA

FDA Therapies Treatment

Behind the new kind of cell therapy that just won FDA approval

BioPharma Drive: Drug Pricing

FEBRUARY 21, 2024

One expert views Amtagvi’s approval as a catalyst for further investment in TIL therapies, akin to how Kymriah’s 2017 clearance buoyed CAR-T treatment.

FDA Approval

FDA Approval Therapies FDA Treatment

Pricey new gene therapies for sickle cell pose access test

BioPharma Drive: Drug Pricing

DECEMBER 8, 2023

Casgevy, the first CRISPR therapy approved by the FDA, will cost $2.2 million, while a competing genetic medicine also cleared Friday is priced at $3.1

Therapies

Therapies FDA

What Do Patients Have to Say about Gene Therapy Trials? An Upcoming FDA Public Meeting to Hear from Patients and Caregivers

FDA Law Blog: Drug Discovery

MARCH 13, 2023

Who better than people living with a condition to inform drug companies, physicians, academics, and the FDA on what it is like to live with their condition, what symptoms most impact their lives, what goes into their decision about whether to participate in a clinical trial, and what kind of treatment effects would be most meaningful to them?

Therapies

Therapies Trials FDA Clinical Trials

FDA Says No to MDMA as Therapy for PTSD

Drugs.com

AUGUST 12, 2024

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. Food and Drug Administration declined to approve it as treatment for.

Therapies

Therapies FDA Treatment Drugs

Lexeo gene therapy shows signs of heart benefit in small study

BioPharma Drive: Drug Pricing

JULY 15, 2024

The company claims the results support exploring the possibility of an accelerated approval, citing flexibility by FDA officials in reviewing rare disease gene therapies.

Therapies

Therapies FDA Disease

Wilson Bryan, former FDA gene therapy leader, joins consulting firm

BioPharma Drive: Drug Pricing

JULY 19, 2023

The agency veteran, who led the Office of Tissues and Advanced Therapies during a boom in gene and cell therapy research, retired in March.

Therapies

Therapies FDA Research

FDA sets advisory meeting date for Brainstorm’s ALS cell therapy

BioPharma Drive: Drug Pricing

JUNE 6, 2023

27 meeting of cell, tissue and gene therapy experts. Having taken the rare step of filing for approval over protest, Brainstorm will get another chance to make a case for its drug NurOwn during a Sept.

Therapies

Therapies FDA Drugs

FDA rejects Abeona cell therapy, asks for more manufacturing data

BioPharma Drive: Drug Pricing

APRIL 23, 2024

The complete response letter for Abeona’s treatment is one of several manufacturing setbacks for cell and gene therapy developers in recent years.

Therapies

Therapies FDA Treatment

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). FRIDAY, June 23, 2023 -- The U.S. The groundbreaking treatment will not be cheap:

FDA Approval

FDA Approval Therapies FDA Treatment

Following FDA rejection, a journal retracts papers on MDMA-assisted therapy

BioPharma Drive: Drug Pricing

AUGUST 12, 2024

Editors at Psychopharmacology cited "unethical conduct" that the study authors didn't disclose when submitting the papers. Lykos Therapeutics says it filed a complaint with a third party to review the way the journal came to its decision.

Therapies

Therapies FDA

What FDA’s Newest Gene Therapy Approval Tells Us About Durability: How Long is Long Enough?

FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

Therapies

Therapies FDA FDA Approval Treatment

FDA Approves First Gene Therapy 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). FRIDAY, June 23, 2023 -- The U.S. The groundbreaking treatment will not be cheap:

FDA Approval

FDA Approval Therapies FDA Treatment

Novartis’ Kisqali gets expanded FDA OK; Mene Pangalos joins Omega Funds

BioPharma Drive: Drug Pricing

SEPTEMBER 18, 2024

The FDA’s clearance could double Kisqali’s breast cancer market. Elsewhere, Novo is exploring new ways to deliver genetic therapies and Bain is funding a growing CDMO.

FDA

FDA Therapies Marketing

Patient death spurs FDA to pause test of Arcellx’s Gilead-partnered cell therapy

BioPharma Drive: Drug Pricing

JUNE 20, 2023

The company said limitations on "bridging treatment" used to hold cancer in check could be to blame, and is working with the FDA to change the study's protocol.

FDA

FDA Therapies Treatment

Orchard follows buyout with FDA approval of rare disease gene therapy

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.

FDA Approval

FDA Approval Therapies Disease FDA

Risk of secondary cancers after CAR-T cell therapy low, according to large study

Science Daily: Pharmacology News

JUNE 12, 2024

In April, the FDA warned of risk of secondary cancers in people receiving CAR-T cell therapy. A large study now finds the risk is low and not related to the CAR-T cells.

Therapies

Therapies FDA

In tone-setting verdict, FDA rejects MDMA as a therapy aid for PTSD

BioPharma Drive: Drug Pricing

AUGUST 9, 2024

The decision to turn down an application from Lykos Therapeutics comes at a pivotal time for psychedelics research, which, after decades of dismissal, has recently gained momentum.

Therapies

Therapies FDA Research

Top FDA official overruled review team in approval of Sarepta’s Duchenne gene therapy

BioPharma Drive: Drug Pricing

JUNE 23, 2023

In a memo made public Friday, Peter Marks, head of the agency office that oversees gene therapies, wrote that he disagreed with other reviewers and saw “compelling” evidence to clear Elevidys.

Therapies

Therapies FDA

Article FDA Thank You New regulatory ‘playbook’ from FDA-supported Bespoke Gene Therapy Consortium aims to accelerate development

Agency IQ

FEBRUARY 12, 2024

BY RACHEL COE, MSC | FEB 7, 2024 10:21 PM CST The Bespoke Gene Therapy Consortium A collaborative effort, the Bespoke Gene Therapy Consortium (BGTC) was launched in October 2021 to “accelerate development of gene therapies for the 30 million Americans who suffer from a rare disease.”

FDA

FDA Therapies Clinical Trials Disease

After an FDA rejection, here’s what’s next in the psychedelics pipeline

BioPharma Drive: Drug Pricing

AUGUST 28, 2024

By rejecting the first MDMA therapy this month, the FDA signaled to the psychedelic drug field that the road to approval isn’t easy.

FDA

FDA Therapies Drugs

Lykos to cut 75% of staff after FDA rejection of MDMA-based therapy

BioPharma Drive: Drug Pricing

AUGUST 15, 2024

The layoffs are part of a larger reorganization that will also see the departure of Lykos’ founder, the psychedelics advocate Rick Doblin.

Therapies

Therapies FDA

Sarepta Duchenne gene therapy wins broader use from FDA

BioPharma Drive: Drug Pricing

JUNE 20, 2024

The decision makes Elevidys available to Duchenne patients at least 4 years of age, despite mixed trial results that have led to skepticism about its effectiveness.

Therapies

Therapies FDA Trials

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.

Therapies

Therapies Clinical Trials FDA Trials

FDA Approves Duvystat, New Oral Treatment for Duchenne Muscular Dystrophy (DMD)

PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. Results from the study that led to the FDA approval appeared in The Lancet Neurology in April 2024 with commentary. Researchers have been working on developing gene therapy for DMD for decades.

FDA Approval

FDA Approval Treatment FDA DNA

BioMarin finally secures FDA approval of hemophilia gene therapy

BioPharma Drive: Drug Pricing

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.

FDA Approval

FDA Approval Therapies FDA

Pfizer wins Canadian OK for hemophilia treatment, its first gene therapy

BioPharma Drive: Drug Pricing

JANUARY 3, 2024

The hemophilia B therapy, which Pfizer will sell in Canada as Beqvez, is also under review in the U.S., where the FDA expects to make a decision by the second quarter.

Therapies

Therapies Treatment FDA

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.

Therapies

Therapies Clinical Trials FDA Trials

Welcome to SRP-RMT – Standardization Comes to Regenerative Medicine Therapies?

FDA Law Blog: Drug Discovery

NOVEMBER 2, 2023

Lewis, Senior Regulatory Device & Biologics Expert — On October 20, 2023, FDA announced the availability of the final guidance authored by CBER titled “Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies.” Tobolowsky & Richard A. It finalized a draft guidance published in 2022.

Therapies

Therapies FDA Production Regulations

Duchenne approval exposes FDA rift over Sarepta gene therapy

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

Trending Sources

First-of-its-kind cell therapy approved by FDA for rare soft tissue cancer

Vertex, CRISPR therapy for sickle cell passes FDA panel test

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

FDA orders new cancer warnings for CAR-T therapies

FDA Advisors Say New Gene Therapy for Sickle Cell Disease, Exa-cel, is Safe

Taysha drops lead gene therapy following FDA feedback

Bristol Myers CAR-T therapy approved by FDA for earlier myeloma use

FDA Approves Landmark Sickle Cell Gene Therapies, Casgevy and Lyfgenia

FDA Approves Landmark Sickle Cell Gene Therapies

With Duchenne decision ahead, FDA’s Marks pushes for speedy gene therapy approvals

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

5 FDA decisions to watch in the second quarter

Rocket gene therapy rejected by FDA over manufacturing

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

Behind the new kind of cell therapy that just won FDA approval

Pricey new gene therapies for sickle cell pose access test

What Do Patients Have to Say about Gene Therapy Trials? An Upcoming FDA Public Meeting to Hear from Patients and Caregivers

FDA Says No to MDMA as Therapy for PTSD

Lexeo gene therapy shows signs of heart benefit in small study

Wilson Bryan, former FDA gene therapy leader, joins consulting firm

FDA sets advisory meeting date for Brainstorm’s ALS cell therapy

FDA rejects Abeona cell therapy, asks for more manufacturing data

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

Following FDA rejection, a journal retracts papers on MDMA-assisted therapy

What FDA’s Newest Gene Therapy Approval Tells Us About Durability: How Long is Long Enough?

FDA Approves First Gene Therapy to Treat Duchenne Muscular Dystrophy

Novartis’ Kisqali gets expanded FDA OK; Mene Pangalos joins Omega Funds

Patient death spurs FDA to pause test of Arcellx’s Gilead-partnered cell therapy

Orchard follows buyout with FDA approval of rare disease gene therapy

Risk of secondary cancers after CAR-T cell therapy low, according to large study

In tone-setting verdict, FDA rejects MDMA as a therapy aid for PTSD

Top FDA official overruled review team in approval of Sarepta’s Duchenne gene therapy

Article FDA Thank You New regulatory ‘playbook’ from FDA-supported Bespoke Gene Therapy Consortium aims to accelerate development

After an FDA rejection, here’s what’s next in the psychedelics pipeline

Lykos to cut 75% of staff after FDA rejection of MDMA-based therapy

Sarepta Duchenne gene therapy wins broader use from FDA

BrainStorm’s ALS therapy not effective, FDA panel finds

FDA Approves Duvystat, New Oral Treatment for Duchenne Muscular Dystrophy (DMD)

BioMarin finally secures FDA approval of hemophilia gene therapy

Pfizer wins Canadian OK for hemophilia treatment, its first gene therapy

BrainStorm’s ALS therapy not effective, FDA panel finds

Welcome to SRP-RMT – Standardization Comes to Regenerative Medicine Therapies?

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