XTalks

JANUARY 2, 2024

For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. Safety and Efficacy of Filsuvez The FDA’s approval of Filsuvez is supported by compelling clinical evidence. Vyjuvek is a gene therapy that employs a herpes simplex virus type 1 (HSV-1) vector.

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Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

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XTalks

MARCH 28, 2024

Italy-based drugmaker Italfarmaco has won US Food and Drug Administration (FDA) approval for its oral medication Duvyzat (givinostat) for the treatment of Duchenne muscular dystrophy (DMD) in patients six years of age and older. The study is one of the largest DMD Phase III trials to date. million last year, of which $131.3

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XTalks

DECEMBER 12, 2023

After receiving US Food and Drug Administration (FDA) approval for Fabhalta (iptacopan) last week for the treatment of the rare blood disorder paroxysmal nocturnal hemoglobinuria (PNH), Novartis presented trial data yesterday showing the drug’s promise in another indication.

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FDA Approval Bacteria Trials Protein 95

Pharmaceutical Technology

SEPTEMBER 5, 2022

The treatment is indicated for CF patients who are homozygous for the F508del mutation (F/F genotype) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Earlier, the therapy had obtained FDA approval for usage in CF patients aged two years and above with two F508del mutation copies.

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Pharmaceutical Technology

JUNE 23, 2023

Sarepta will now need to complete a confirmatory trial to prove the therapy improves physical function and mobility.

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XTalks

MAY 17, 2023

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio.

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XTalks

JANUARY 3, 2024

Wainua is the only FDA-approved drug for the treatment of ATTRv-PN that can be self-administered via an auto-injector. Polydefkis, professor of Neurology at Johns Hopkins University School of Medicine and an investigator in the Phase III NEURO-TTRansform study on which Wainua’s FDA approval was grounded on, in the news release.

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Pharmaceutical Technology

APRIL 18, 2023

The US Food and Drug Administration (FDA) has granted approval for the expanded indication of AbbVie ’s Qulipta (atogepant) as a preventive treatment for chronic migraines in adult patients. Qulipta received approval to treat migraine across frequencies, including both episodic and chronic.

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FDA Approval Botox Gene Trials 130

Drug Discovery World

JUNE 12, 2023

The US Food and Drug Administration (FDA) has approved Minoryx Therapeutics’ Phase III clinical trial (CALYX) of leriglitazone, to treat adult male X-linked Adrenoleukodystrophy (X-ALD) patients with cerebral Adrenoleukodystrophy (cALD).

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Drug Discovery World

SEPTEMBER 20, 2022

SKYSONA has become the first FDA approved therapy to slow the progression of neurologic dysfunction in boys with cerebral adrenoleukodystrophy (CALD). As a condition of the SKYSONA Accelerated Approval, the manufacturer will provide confirmatory long-term clinical data to the FDA.

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XTalks

FEBRUARY 6, 2023

GlaxoSmithKline LLC (GSK) recently announced that the US Food and Drug Administration (FDA) approved Jesduvroq (daprodustat), a new once-a-day oral treatment for anemia due to chronic kidney disease (CKD). The approval is currently only for adult patients who have been undergoing dialysis for at least four months.

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XTalks

DECEMBER 27, 2022

Ferring Pharmaceuticals announced that the US Food and Drug Administration (FDA) approved Adstiladrin (nadofaragene firadenovec-vncg) for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

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Gene Therapy FDA Approval Gene Clinical Trials 52

XTalks

AUGUST 31, 2022

In this episode, Ayesha discussed the FDA approval of Axsome Therapeutics’ rapid-acting oral treatment Auvelity for the treatment of major depressive disorder (MDD). The approval makes Auvelity the first and only rapid-acting oral medication for depression, and the first and only oral NMDA receptor antagonist approved for MDD.

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XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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pharmaphorum

MAY 13, 2021

The first UK patient has enrolled on Pfizer’s phase 3 trial for its Duchenne muscular dystrophy (DMD) gene therapy. The first patient was recruited in The Newcastle upon Tyne Hospitals NHS Foundation Trust, one of three UK sites for the clinical trial and part of 55 globally, across 15 countries.

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Drug Discovery World

FEBRUARY 19, 2024

“The approval of Amtagvi represents the culmination of scientific and clinical research efforts leading to a novel T cell immunotherapy for patients with limited treatment options.” Amtagvi is the first FDA-approved tumour-derived T cell immunotherapy. Among patients who were responsive to the treatment, 56.5%, 47.8%

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Drug Discovery World

OCTOBER 18, 2023

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies. Adeno-associated virus (AAV) vector-based gene therapies hold exceptional promise across a range of disease areas.

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XTalks

JUNE 28, 2023

Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). Additionally, the FDA said a “clinical benefit of Elevidys, including improved motor function, has not been established.”

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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., For the U.S.

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pharmaphorum

AUGUST 18, 2022

bluebird bio’s Zynteglo has become the first cell-based gene therapy to be approved in the US, getting the nod from the FDA as a treatment for patients with beta thalassaemia who require regular blood transfusions. An FDA advisory committee recommended approval of Zynteglo on the strength of that data in June.

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XTalks

NOVEMBER 10, 2023

cTTP is a very rare, inherited and life-threatening blood clotting disorder caused by a disease-causing mutation in the ADAMTS13 (A disintegrin and metalloproteinase with thrombospondin motifs 13) gene, which encodes the ADAMTS13 enzyme that regulates blood clotting by cleaving the von Willebrand factor (VWF) protease.

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The Pharma Data

AUGUST 24, 2020

The FDA has approved a request from American Gene Technologies to begin a clinical study into its HIV gene therapy. The treatment is being researched by scientists collaborating from American Gene Technologies, the Laboratory of Immunoregulation and the National Institute of Allergy and Infectious Diseases.

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Gene Therapy Gene FDA Approval Trials 52

XTalks

APRIL 24, 2024

ImmunityBio’s Anktiva (N-803, or nogapendekin alfa inbakicept-pmln) along with the Bacillus Calmette-Guérin (BCG) vaccine has won US Food and Drug Administration (FDA) approval for the treatment of non-muscle invasive bladder cancer (NMIBC). months in the KEYNOTE-057 trial.

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XTalks

DECEMBER 10, 2021

Biohaven Pharmaceuticals revealed positive top line results from a late-stage trial which show that its migraine medication Zavegepant provides “ultra-rapid” pain relief in just 15 minutes. Zavegepant is an antibody drug that prevents binding of the calcitonin gene-related peptide (CGRP) to the CGRP receptor.

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XTalks

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB). It has been modified so that it cannot replicate in normal cells.

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Drug Discovery World

AUGUST 14, 2023

Janssen’s Akeega (niraparib and abiraterone acetate) is now available in the US for adult patients with deleterious or suspected deleterious BRCA-positive metastatic castration-resistant prostate cancer (mCRPC) following FDA approval. Approximately 10-15% of patients with mCRPC have BRCA gene alterations.

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XTalks

SEPTEMBER 12, 2022

After approvals in Japan and Europe, Sanofi’s Xenpozyme has secured one from the US Food and Drug Administration (FDA) for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. Patients in the trial received Xenpozyme or placebo for 52 weeks.

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Drug Discovery World

OCTOBER 12, 2023

Samulski’s graduate work demonstrated the first substrate for adeno-associated viruses (AAV) as a viral vector and culminated in the first US patent involving non-AAV genes inserted into AAV. Dr Samulski’s discoveries in AAV played a profound role in shaping the evolution of the gene therapy field.

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Drug Discovery World

OCTOBER 12, 2023

Samulski’s graduate work demonstrated the first substrate for adeno-associated viruses (AAV) as a viral vector and culminated in the first US patent involving non-AAV genes inserted into AAV. Dr Samulski’s discoveries in AAV played a profound role in shaping the evolution of the gene therapy field.

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XTalks

JUNE 30, 2023

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII).

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XTalks

JUNE 17, 2022

Eli Lilly’s Olumiant (baricitinib) has won US Food and Drug Administration (FDA) approval as the first systemic treatment for severe alopecia areata (AA), an autoimmune disorder that leads to patchy baldness. The Olumiant approval is therefore a big win as it helps fulfill a significant unmet need in the area.

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FDA Approval Gene Expression Cosmetics Trials 97

pharmaphorum

JUNE 10, 2022

The FDA may have safety concerns abut bluebird bio’s gene therapy for rare, fatal disease cerebral adrenoleukodystrophy (CALD), but its advisors believe its benefits far outweigh the risks. bluebird is also conducting the phase 3 ALD-104 trial of eli-cel in CALD, which is due to generate results in 2024.

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XTalks

APRIL 11, 2024

After backing from the US Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee (ODAC) last month, Johnson and Johnson (J&J) and Bristol Myers Squibb (BMS) scored FDA approvals for the use of their B-cell maturation antigen (BCMA)-targeting CAR T-cell therapies as earlier line treatment options for multiple myeloma.

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FDA Law Blog

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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XTalks

JUNE 2, 2021

KRAS mutations have previously been considered ‘undruggable’ targets, but they have been a big focus in cancer research as they are one of the most common gene mutations across all cancers. The sotorasib approval is therefore a major breakthrough in the world of cancer and targeted therapies.

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XTalks

JANUARY 26, 2023

The US Food and Drug Administration (FDA) recently granted approval for the Bruton tyrosine kinase inhibitor (BTKi) Brukinsa (zanubrutinib) based on the results from two Phase III clinical trials. Brukinsa is now approved to treat adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).

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