Pharmaceutical Technology

MAY 22, 2023

It is claimed to be both the first re-dosable gene therapy and the first and only FDA-approved treatment for both recessive and dominant types of DEB, a rare and serious genetic disease affecting the skin and mucosal tissues.

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FDA Approval Gene Therapy Genetic Disease Gene 130

XTalks

APRIL 3, 2024

Related: Novartis’ Fabhalta Gets FDA Approval for Rare Complement Blood Disorder The FDA’s green light for Voydeya comes three months after the Japanese Ministry of Health, Labour and Welfare (JHLW) became the first regulator in the world to back the therapy. AstraZeneca is also venturing into cell therapy and genetic medicine.

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Pharma Mirror

MARCH 10, 2021

A post-pandemic pharma report from CPhI predicts the next few years will deliver strong growth in pharma innovation, with oncology and rare diseases potentially the biggest beneficiaries of positive covid legacies – including accelerated development timelines, remote trials and new technologies in genetic and target screening.

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FDA Approval Genetics Trials Development 130

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. Approval of Wainua represents a meaningful advancement in treatment, one that gives those who are living with transthyretin-mediated amyloid polyneuropathy help managing the disease,” said Michael J.

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FDA Approval Protein RNA Clinical Trials 105

Pharmaceutical Technology

MAY 26, 2023

Krystal Biotech has received approval from the US Food and Drug Administration for topical gene therapy VYJUVEK to treat dystrophic epidermolysis bullosa (DEB) in adults and in children aged six months and above. VYJUVEK is designed to address the underlying genetic cause of the disease.

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Gene Therapy Gene FDA Approval Manufacturing 264

XTalks

JANUARY 2, 2024

For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. a biotech company specializing in the development and commercialization of genetic medicines for rare diseases, announced FDA approval for Vyjuvek for the treatment of DEB.

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FDA Approval Gene Therapy Gene Trials 111

Pharmaceutical Technology

FEBRUARY 15, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Genetically modified animal models. Regeneron Pharmaceuticals is one of the leading patent filers in the generation of genetically modified animal models.

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pharmaphorum

MARCH 26, 2024

Nanoscope Therapeutics is on the brink of filing for FDA approval of what could be the first gene therapy for incurable eye disease retinitis pigmentosa (RP) that can be used regardless of underlying genetic mutations.

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Gene Therapy Gene FDA Approval Genetics 127

BioSpace

OCTOBER 22, 2023

The label for the achondroplasia drug, which promotes endochondral bone growth, now covers children under five years of age with the rare genetic disease causing the most common form of dwarfism.

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FDA Approval Genetic Disease Genetics Drugs 108

XTalks

JUNE 20, 2022

Amvuttra’s FDA approval is based on positive data from the global, randomized, open-label, multicenter HELIOS-A Phase III study from a period of nine months. The condition has very limited treatment options. Amvuttra is administered via subcutaneous injection four times a year or once every three months (quarterly).

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FDA Approval Protein Genetics RNA 98

Drug Discovery World

JUNE 23, 2023

The accelerated approval follows a vote of support from the FDA Cellular, Tissue and Gene Therapies Advisory Committee and the granting of priority review status. The post FDA approves first gene therapy for Duchenne muscular dystrophy appeared first on Drug Discovery World (DDW).

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Gene Therapy FDA Approval Gene Protein 97

The Pharma Data

DECEMBER 14, 2020

FDA Approves Genetically Engineered Pigs for Food, Possible Medical Use. 14, 2020 — The first genetically engineered pigs for use as food or for potential future biomedical use — such as transplantation — in humans have been approved by the U.S. Professional. MONDAY, Dec.

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Genetic Engineering Engineer FDA Approval Genetics 52

XTalks

SEPTEMBER 14, 2022

In this episode, Ayesha discussed the FDA approval of Sanofi’s enzyme replacement therapy Xenpozyme for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD), a rare genetic lysosomal storage disease, in adults and pediatric patients.

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Life Science FDA Approval Insulin Drugs 90

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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BioSpace

MARCH 13, 2024

Wednesday’s FDA approval expands Mirum’s Livmarli into the rare genetic disorder that causes progressive liver disease. The biotech has also filed a supplemental New Drug Application for a higher dose of the drug and allowing its use in younger patients.

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FDA Approval Genetics Drugs 109

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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FDA Approval Genetics Trials Regulation 98

Drug Discovery World

MAY 29, 2023

It is approved for treating wounds in patients six months of age and older with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene. Vyjuvek is the first FDA-approved gene therapy treatment for DEB, a rare and serious genetic skin disorder,” said Peter Marks, Director of the FDA’s Center for Biologics Evaluation and Research.

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Gene Therapy FDA Approval Gene Genetics 52

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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Gene Therapy FDA Approval Gene Gene Editing 52

BioSpace

JULY 7, 2022

Last year, the FDA's Center for Drug Evaluation and Research approved 50 drugs. A new study found that 33, or 66% of them, were supported with genomic data.

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FDA Approval Genetics Genome Genomics 86

XTalks

DECEMBER 13, 2023

This approval also marks bluebird’s third ex vivo gene therapy approved by the FDA for a rare genetic disease and second FDA approval for an inherited hemoglobin disorder, cementing our position as a gene therapy leader.”

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Gene Therapy FDA Approval Gene In-Vivo 98

XTalks

JANUARY 26, 2023

This is the fourth US FDA approval that BeiGene has received for Brukinsa since 2019. Diagnosis is confirmed by a differential complete blood count and genetic testing. Treatment selection is determined by age, disease severity, comorbidities and the presence or absence of certain genetic mutations.

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FDA Approval Life Science Clinical Trials Clinical Trials 97

XTalks

JULY 19, 2021

Phosphorus, a leading preventative genomics company, has developed the first comprehensive preventative genetic test for consumers. The test is called GeneCompass and features medical-grade technology to provide a holistic assessment of genetic health and wellness. The Phosphorus GeneCompass test has a list price of $249.

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Genetics Genome Genomics Gene 98

XTalks

NOVEMBER 10, 2023

Today, we are proud to further support the rare disease community by delivering Adzynma as the first FDA-approved therapeutic option for people with cTTP.” In 2022, the FDA approved 20 orphan drugs out of the 37 novel drugs approved by the FDA’s Center for Drug Evaluation and Research (CDER).

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FDA Approval Gene Therapy Gene Clinical Trials 59

XTalks

JUNE 23, 2022

In this episode, Ayesha and the team talked about the FDA approval of Eli Lilly’s JAK inhibitor Olumiant for the treatment of alopecia areata, also commonly just known as alopecia. The drug has become the first approved systemic treatment for the autoimmune disorder that causes patchy hair loss.

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Life Science FDA Approval Protein Drugs 52

Drug Discovery World

JANUARY 23, 2023

Armed with this understanding, the researchers further demonstrated that the genetic inhibition of DGAT1 suppresses radioresistance. As an FDA-approved oral drug, the side-effects of cladribine are quite manageable and it has been well evaluated for pharmacokinetics.

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FDA Approval Drugs In-Vivo In-Vitro 52

Roots Analysis

JANUARY 19, 2024

Introduction In a strategic move to accelerate the development and launch of genetic medicines, BridgeBio Pharma has successfully secured up to USD 1.25 In a deal worth USD 500 million in cash, BridgeBio Pharma has committed to providing the investors with a 5% royalty on worldwide net sales of its drug, acoramidis, pending FDA approval.

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XTalks

MARCH 10, 2023

This US Food and Drug Administration (FDA)-approved drug was also awarded a rare pediatric disease priority review voucher. In the MOXIe Part 2 trial, 82 patients with Friedreich’s ataxia (as confirmed by genetic testing) were randomized 1:1 to receive either 150 mg omaveloxolone or placebo.

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Medical Xpress

DECEMBER 13, 2022

New research has confirmed a strong link between four genetic mutations and progressive multifocal leukoencephalopathy (PML), a rare but often fatal brain infection that can be triggered by dozens of FDA-approved drugs.

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FDA Approval Research Gene Genetics 98

BioSpace

APRIL 21, 2021

The approval was specifically for patients with recurrent or advanced endometrial cancer who had progressed on or after previous treatment with platinum-based chemotherapy and whose cancers have a dMMR genetic anomaly.

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FDA Approval Genetics 98

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 Approval Manufacturing Trials Clinical Trials 52

BioPharma Reporter

AUGUST 18, 2022

Food and Drug Administration (FDA) has approved bluebird bioâs Zynteglo (betibeglogene autotemcel), also known as beti-cel: a one-time gene therapy custom-designed to treat the underlying genetic cause of beta thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions.

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Gene Therapy Gene FDA Approval Genetics 105

Pharmaceutical Commerce

JANUARY 24, 2024

Treatments required to have updated warnings include all FDA-approved CAR T-cell therapies and licensed BCMA-directed and CD19-directed genetically modified autologous CAR T-cell immunotherapies.

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FDA Approval Licensing Licensing Genetics 52

Medical Xpress

NOVEMBER 23, 2022

People with one form of the genetic blood disorder hemophilia now have a one-time treatment with a $3.5 million price tag.

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Gene Therapy FDA Approval Gene Genetics 74

pharmaphorum

SEPTEMBER 1, 2022

Sanofi now has approval on both sides of the Atlantic for Xenpozyme – its enzyme replacement therapy for rare disease acid sphingomyelinase deficiency (ASMD) – after getting a green light from the US FDA. The post Xenpozyme is first FDA-approved drug for rare disease ASMD appeared first on.

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FDA Approval Drugs Genetics Trials 52

pharmaphorum

NOVEMBER 24, 2020

The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. The post FDA approves Alnylam’s ultra-rare disease drug Oxlumo appeared first on.

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FDA Approval Gene Silencing Drugs RNA 52

pharmaphorum

MARCH 29, 2021

Abecma’s review was delayed after the FDA rejected a first filing in May last year, but it’s hoped that the drug could be a significant step forward in the treatment of this incurable condition. The delay was caused by coronavirus restrictions delaying the site inspections necessary for approval.

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FDA Approval Genetics Protein Antibody 61

The Pharma Data

AUGUST 9, 2021

“Pompe disease is a rare genetic disease that causes premature death and has a debilitating effect on people’s lives,” said Janet Maynard, M.D., deputy director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research.

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FDA Approval Genetic Disease Genetics Drugs 52