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 endorses speedy approval path for Regenxbio Duchenne gene therapy

BioPharma Drive: Drug Pricing

NOVEMBER 18, 2024

The agency's openness to a targeted pivotal study shows it’s still willing to consider accelerated clearance for Duchenne gene therapies despite questions about their effectiveness.

Therapies

Therapies FDA

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 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

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 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 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

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

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

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

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

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

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

Sarepta scraps a Duchenne drug as gene therapy sales rise

BioPharma Drive: Drug Pricing

NOVEMBER 7, 2024

Elevidys sales have increased since the FDA made a controversial choice to expand the therapy's use. Now, Sarepta is abandoning a successor to its drug Exondys 51, citing an “evolving" treatment landscape.

Therapies

Therapies Drugs 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

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

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

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

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

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

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

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

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

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

First Duchenne gene therapy approved by FDA for young children

BioPharma Drive: Drug Pricing

JUNE 22, 2023

The conditional clearance for Sarepta’s Elevidys in 4- and 5-year-olds is a milestone for research into the deadly disease, and raises the stakes of an ongoing trial that could prove how well it works.

Therapies

Therapies FDA Trials Disease

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

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

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

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

Orchard nears FDA decision on rare disease gene therapy

BioPharma Drive: Drug Pricing

SEPTEMBER 18, 2023

Three years after gaining European approval, Libmeldy is now under U.S. review with a deadline set for March.

Therapies

Therapies Disease FDA

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.

Therapies

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

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

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

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.

FDA

FDA Therapies Treatment

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

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

5 FDA decisions to watch in the third quarter

BioPharma Drive: Drug Pricing

JULY 3, 2024

Multibillion-dollar buyouts from Bristol Myers Squibb and Gilead could yield new drugs for brain and liver diseases, while a new cell therapy may reach market.

FDA

FDA Therapies Disease Marketing

FDA Approves First Gene Therapy for Severe Hemophilia A, Roctavian

Drugs.com

JUNE 30, 2023

Food and Drug Administration on Thursday approved a costly single-dose gene therapy for patients with severe hemophilia A. FRIDAY, June 30, 2023 -- The U.S. The treatment, Roctavian (valoctocogene roxaparvovec), will cost $2.9 million for a single.

FDA Approval

FDA Approval Therapies FDA Treatment

Bristol Myers faces FDA delay on cancer cell therapy decision

BioPharma Drive: Drug Pricing

NOVEMBER 20, 2023

The regulator plans to convene an advisory panel to discuss an expanded indication for Abecma, presenting another hurdle for Bristol Myers and partner 2seventy bio.

Therapies

Therapies FDA Regulations

Sarepta gene therapy for Duchenne misses main goal of key study

BioPharma Drive: Drug Pricing

OCTOBER 30, 2023

Yet Sarepta said it still plans to ask the FDA to expand regulatory clearance of Elevidys, which was granted an accelerated approval by the FDA in June.

Therapies

Therapies FDA

Vertex gearing up for launch as sickle cell therapy review advances

BioPharma Drive: Drug Pricing

OCTOBER 26, 2023

Executives at the biotech say they’re trying to get ahead of the payer and production challenges that will face their gene editing treatment exa-cel, which is now under FDA review.

Therapies

Therapies FDA Treatment Production

Duchenne approval exposes FDA rift over Sarepta gene therapy

FDA endorses speedy approval path for Regenxbio Duchenne gene therapy

Trending Sources

Vertex, CRISPR therapy for sickle cell passes FDA panel test

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

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

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

FDA orders new cancer warnings for CAR-T therapies

FDA Approves Landmark Sickle Cell Gene Therapies, Casgevy and Lyfgenia

5 FDA decisions to watch in the second quarter

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

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

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

Lexeo gene therapy shows signs of heart benefit in small study

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

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

Sarepta scraps a Duchenne drug as gene therapy sales rise

Pricey new gene therapies for sickle cell pose access test

FDA Approves Landmark Sickle Cell Gene Therapies

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

BioMarin finally secures FDA approval of hemophilia gene therapy

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

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

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

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

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

FDA Says No to MDMA as Therapy for PTSD

First Duchenne gene therapy approved by FDA for young children

FDA Approves First Gene Therapy to Treat Duchenne Muscular Dystrophy

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

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

BrainStorm’s ALS therapy not effective, FDA panel finds

Orchard nears FDA decision on rare disease gene therapy

FDA investigating cancer risk linked to CAR-T cell therapy

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

Taysha drops lead gene therapy following FDA feedback

BrainStorm’s ALS therapy not effective, FDA panel finds

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

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

Rocket gene therapy rejected by FDA over manufacturing

5 FDA decisions to watch in the third quarter

FDA Approves First Gene Therapy for Severe Hemophilia A, Roctavian

Bristol Myers faces FDA delay on cancer cell therapy decision

Sarepta gene therapy for Duchenne misses main goal of key study

Vertex gearing up for launch as sickle cell therapy review advances

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