FDA, Therapies and Treatment - Drug Discovery Digest

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 FDA Drugs Treatment

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. A gene-based treatment would have to alter many cells to exert a noticeable effect.

FDA Approval

FDA Approval Treatment FDA DNA

FDA Returns Disappointing News for ALS Stem Cell Therapy

PLOS: DNA Science

OCTOBER 19, 2023

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.

Therapies

Therapies FDA Clinical Trials DNA

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

Insights into cellular therapies for cancer treatment

Drug Target Review

FEBRUARY 21, 2024

Stem cell transplants have saved patients’ lives time and time again, which led us to launch our own Stem Cell Transplant and Cellular Therapy Program. Regarding CAR-T cell therapy, could you highlight the promising findings that have been identified in the early stages of drug discovery, as well as potential challenges?

Therapies

Therapies Treatment Vaccine FDA Approval

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 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

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

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.

Clinical Development

Clinical Development Production Clinical Trials Treatment

Advancing CAR-T therapy: how CD5 modulation is shaping cancer treatment

Drug Target Review

AUGUST 29, 2024

What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies? MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs.

Therapies

Therapies Treatment Engineering Molecular Biology

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

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.

Therapies

Therapies FDA Treatment Drugs

Article FDA: In a first, FDA tackles treatments for stimulant use disorder

Agency IQ

OCTOBER 6, 2023

In a first, FDA tackles treatments for stimulant use disorder A new draft guidance from the FDA gives developers a roadmap to advance the development of novel therapies to address stimulant use disorders.

FDA

FDA Treatment Therapies Drugs

FDA Approval of New Therapy, Duvyzat, for Duchenne Muscular Dystrophy Represents Several Meaningful Firsts

FDA Law Blog: Drug Discovery

MARCH 26, 2024

Sasinowski — On March 21st, FDA announced the approval of the first nonsteroidal therapy for the treatment of Duchenne Muscular Dystrophy (DMD) (FDA press release available here ). By Charles G. Raver & James E. Valentine & Frank J. Hyman, Phelps & McNamara, P.C.’s

FDA Approval

FDA Approval Therapies FDA Trials

New gene delivery vehicle shows promise for human brain gene therapy

Broad Institute

MAY 16, 2024

Gene therapy could potentially treat a range of severe genetic brain disorders, which currently have no cures and few treatment options. Since we came to the Broad we’ve been focused on the mission of enabling gene therapies for the central nervous system,” said Deverman, senior author on the study. “If

Therapies

Therapies FDA Approval Engineering Disease

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.

FDA Approval

FDA Approval Therapies FDA Treatment

The Significance of the MHRA Approval and Upcoming FDA Review of the First Gene Editing Treatment

Conversations in Drug Development Trends

NOVEMBER 27, 2023

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow.

Treatment

Treatment FDA Clinical Trials Therapies

FDA Approves New Treatment for Advanced Melanoma, Amtagvi

Drugs.com

FEBRUARY 19, 2024

Food and Drug Administration has approved a novel treatment for advanced melanoma, the most deadly form of skin cancer. becomes the first cellular therapy approved to. MONDAY, Feb. 19, 2024 -- The U.S. Amtagvi, made by Iovance Biotherapeutics Inc.,

FDA Approval

FDA Approval Treatment FDA Therapies

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

FDA Approval

FDA Approval Therapies FDA Treatment

At Long Last, FDA Unveils Plan for Rare Disease Innovation Hub

FDA Law Blog: Drug Discovery

JULY 17, 2024

Importantly, the Hub is intended to establish a new model within FDA, which leverages cross-Agency expertise in providing guidance and conducting reviews for products for rare disease populations. By Sarah Wicks & James E. Valentine & Frank J. those reviewed by the CDER Division of Rare Diseases and Medical Genetics).

FDA

FDA Disease Clinical Pharmacology Science

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 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

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 Approves New Treatment for Advanced Melanoma

Drugs.com

FEBRUARY 19, 2024

Food and Drug Administration has approved a novel treatment for advanced melanoma, the most deadly form of skin cancer.Amtagvi, made by Iovance Biotherapeutics Inc., becomes the first cellular therapy approved to. MONDAY, Feb. 19, 2024 -- The U.S.

FDA Approval

FDA Approval Treatment FDA Therapies

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

Scaling Phage Therapy

Codon

FEBRUARY 13, 2024

Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.

Therapies

Therapies DNA Virus Clinical Trials

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

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 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. The treatment, Roctavian (valoctocogene roxaparvovec), will cost $2.9 FRIDAY, June 30, 2023 -- The U.S. million for a single.

FDA Approval

FDA Approval Therapies FDA Treatment

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

Lucanthone, a Potential PPT1 inhibitor, Perturbs Stemness, Reduces Tumor Microtube Formation and Slows the Growth of Temozolomide-Resistant Gliomas in Vivo [Chemotherapy, Antibiotics, and Gene Therapy]

ASPET

JANUARY 31, 2024

Despite the standard of care therapy which includes surgical resection, temozolomide chemotherapy, radiation and the newly added tumor treating fields, median survival remains only ~20 months. Our results suggest that lucanthone may function as a potential treatment option for GBM tumors that are not amenable to TMZ treatment.

Therapies

Therapies FDA Approval Laboratories Treatment

Sloane’s Journey with ASO Therapy

KIF1A

APRIL 25, 2024

She is scheduled to be the 2nd patient in the world to receive ASO therapy for KAND. Her mother, Megan, has offered to share their experience and journey with everyone to help us understand the process and to share in their hopes, fears, challenges, and successes that come along with ASO treatment. Hello KIF1A Community!

Therapies

Therapies FDA Approval FDA Treatment

FDA approves new treatment for Pompe disease

The Pharma Data

AUGUST 9, 2021

deputy director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research. “Today’s approval brings patients with Pompe disease another enzyme replacement therapy option for this rare disease.

FDA Approval

FDA Approval FDA Disease Treatment

FDA staff outline shortcomings of Lykos data for MDMA therapy

BioPharma Drive: Drug Pricing

MAY 31, 2024

Ahead of an advisory panel meeting, agency staff identified several sticking points in the clinical results backing Lykos’ ecstasy-based treatment for PTSD.

Therapies

Therapies FDA Treatment

AdComms – Is FDA Getting Less Advice?

Eye on FDA

AUGUST 4, 2021

As more drugs are being approved, is FDA getting less advice than in the past? FDA maintains a vast network of outside advisors to provide input and counsel to the agency related to decisions on policy as well as product approvals. There are 31 advisory committees on matters ranging from food to medical devices to pediatric care.

FDA

FDA Clinical Pharmacology FDA Approval Therapies

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.

FDA Approval

FDA Approval Treatment FDA Therapies

FDA Advisors to Weigh New Gene Therapy for Sickle Cell Anemia

Drugs.com

OCTOBER 31, 2023

30, 2023 -- Patients with sickle cell disease may soon have two new treatments to try. Food and Drug Administration advisory committee will weigh the merits of a new gene therapy for the painful, inherited. MONDAY, Oct. On Tuesday, a U.S.

Therapies

Therapies FDA Treatment Disease

Topical Gene Therapy FDA-Approved for Severe Skin Disease, Dystrophic Epidermolysis Bullosa

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. DEB has been a candidate for a gene therapy since 2002.

FDA Approval

FDA Approval Therapies FDA Disease

FDA grants efanesoctocog alfa Breakthrough Therapy designation for hemophilia A

The Pharma Data

JUNE 1, 2022

The United States Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to efanesoctocog alfa (BIVV001) for the treatment of people with hemophilia A, a rare and life-threatening bleeding disorder, based on data from the pivotal XTEND-1 P hase 3 stud y.

Therapies

Therapies FDA Science Treatment

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

Sarepta scraps a Duchenne drug as gene therapy sales rise

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

Trending Sources

FDA Returns Disappointing News for ALS Stem Cell Therapy

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

Insights into cellular therapies for cancer treatment

Vertex, CRISPR therapy for sickle cell passes FDA panel test

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

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

FDA Approves Landmark Sickle Cell Gene Therapies, Casgevy and Lyfgenia

FDA Approves Landmark Sickle Cell Gene Therapies

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

Advancing CAR-T therapy: how CD5 modulation is shaping cancer treatment

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

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

FDA Says No to MDMA as Therapy for PTSD

Article FDA: In a first, FDA tackles treatments for stimulant use disorder

FDA Approval of New Therapy, Duvyzat, for Duchenne Muscular Dystrophy Represents Several Meaningful Firsts

New gene delivery vehicle shows promise for human brain gene therapy

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

The Significance of the MHRA Approval and Upcoming FDA Review of the First Gene Editing Treatment

FDA Approves New Treatment for Advanced Melanoma, Amtagvi

FDA Approves First Gene Therapy to Treat Duchenne Muscular Dystrophy

At Long Last, FDA Unveils Plan for Rare Disease Innovation Hub

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

FDA rejects Abeona cell therapy, asks for more manufacturing data

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

FDA Approves New Treatment for Advanced Melanoma

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

Scaling Phage Therapy

FDA investigating cancer risk linked to CAR-T cell therapy

BrainStorm’s ALS therapy not effective, FDA panel finds

FDA Approves First Gene Therapy for Severe Hemophilia A, Roctavian

BrainStorm’s ALS therapy not effective, FDA panel finds

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

Lucanthone, a Potential PPT1 inhibitor, Perturbs Stemness, Reduces Tumor Microtube Formation and Slows the Growth of Temozolomide-Resistant Gliomas in Vivo [Chemotherapy, Antibiotics, and Gene Therapy]

Sloane’s Journey with ASO Therapy

FDA approves new treatment for Pompe disease

FDA staff outline shortcomings of Lykos data for MDMA therapy

AdComms – Is FDA Getting Less Advice?

Nestle gives up on peanut allergy treatment after sluggish sales

FDA Advisors to Weigh New Gene Therapy for Sickle Cell Anemia

Topical Gene Therapy FDA-Approved for Severe Skin Disease, Dystrophic Epidermolysis Bullosa

FDA grants efanesoctocog alfa Breakthrough Therapy designation for hemophilia A

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

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