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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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.

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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.

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In a first, FDA tackles treatments for stimulant use disorder A new draft guidance from the FDA gives developers a roadmap to advance the development of novel therapies to address stimulant use disorders.

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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.

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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.,

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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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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.

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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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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.

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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.

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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?

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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.

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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.

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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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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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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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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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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.

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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!

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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.

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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.

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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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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.

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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.”

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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. While stimulant use disorder is increasing, there are currently no FDA-approved medications.

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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.

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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.

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The Pharma Data

FEBRUARY 24, 2022

Designation is supported by Phase II efficacy and safety data that will be presented at ATS 2022 Boehringer Ingelheim plans to study this novel investigational therapy in patients with progressive fibrosing interstitial lung diseases. The FDA Breakthrough Therapy designation is supported by data collected to date.

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The Pharma Data

JUNE 2, 2023

Food and Drug Administration (FDA) for the treatment of adult patients with deleterious or suspected deleterious BRCA -mutated ( BRCA m) metastatic castration-resistant prostate cancer (mCRPC). Patients should be selected for therapy based on an FDA-approved companion diagnostic for LYNPARZA. In the U.S., In the U.S.,

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The Pharma Data

DECEMBER 15, 2020

Food and Drug Administration (FDA) granted Novartis ’ experimental drug iptacopan Breakthrough Therapy designation in paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disorder. Iptacopan also demonstrated positive interim results as a monotherapy treatment in anti-C5 naïve PNH patients.

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BioPharma Drive: Drug Pricing

AUGUST 15, 2023

The agency had paused testing after a patient death, but is now permitting more types of bridging treatment to help keep participants’ disease at bay.

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Broad Institute

SEPTEMBER 13, 2023

Developing diagnostics and treatments By Rose Circeo September 13, 2023 Breadcrumb Home Developing diagnostics and treatments At the Broad Institute, researchers are revealing deep biological insights about the causes of disease, which are leading to new ways to monitor, diagnose, and treat patients with greater precision.

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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.

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Eye on FDA

MARCH 7, 2024

Back in December 2023, FDA announced intention in the Federal Register and in a press release to form a new FDA Advisory Committee to be called the Genetic Metabolic Diseases Advisory Committee (GeMDAC). GeMDAC at this time does not have any members appointed.

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