XTalks

DECEMBER 18, 2024

The therapy is approved to be used in combination with glucocorticoid replacement therapies (steroids) to control androgen levels in adults and pediatric patients four years of age and older with classic CAH.

Genetics 116

Genetics Hormones Trials Drugs 116

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.

Gene Therapy 264

Gene Therapy Gene FDA Approval Manufacturing 264

Bio Pharma Dive

FEBRUARY 24, 2025

Mirum gained the drug, which it will sell as Ctexli for adults with cerebrotendinous xanthomatosis, in a 2023 deal with Travere Therapeutics.

Genetic Disease 143

Genetic Disease FDA Approval Genetics Drugs 143

XTalks

FEBRUARY 25, 2025

Mirum Pharmaceuticals, a biotech innovator known for developing therapies for rare metabolic disorders, now has FDA approval for its new treatment, Ctexli (chenodiol) tablets the first and only medication approved for cerebrotendinous xanthomatosis (CTX) in adults.

FDA Approval 59

FDA Approval Gene Therapy Gene Genetics 59

BioPharma Reporter

NOVEMBER 19, 2024

PTC Therapeutics has received FDA approval for a gene therapy to treat AADC deficiency, a rare genetic disorder that impairs the normal function of neurons.

Gene Therapy 114

Gene Therapy FDA Approval Gene Genetics 114

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.

FDA Approval 130

FDA Approval Genetics Trials Development 130

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. Duvyzat received Priority Review, Orphan Drug and Rare Pediatric Disease designations from the FDA.

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FDA Approval Genetics Gene Therapy Gene 105

XTalks

JANUARY 21, 2025

TriClip G4 System Manufacturer/developer : Abbott Medical Date of FDA approval : April 1, 2024 Approved for : Tricuspid regurgitation (TR). Date of FDA approval : March 29, 2024 Approved for : To detect exposure to human parvovirus B19. TriClip size comparison photo. Photo courtesy of Abbott Medical.

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FDA Approval Manufacturing Development RNA 105

BioPharma Reporter

NOVEMBER 19, 2024

PTC Therapeutics has received FDA approval for a gene therapy to treat AADC deficiency, a rare genetic disorder that impairs the normal function of neurons.

Gene Therapy 99

Gene Therapy FDA Approval Gene Genetics 99

XTalks

JANUARY 6, 2025

The US Food and Drug Administration (FDA) has approved Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), a next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator, to treat cystic fibrosis (CF) in patients aged six years and older with at least one F508del mutation or another responsive CFTR mutation.

Gene Therapy 105

Gene Therapy Protein Genetic Disease Gene 105

Pharmaceutical Technology

JUNE 14, 2023

Ipsen has received US Food and Drug Administration (FDA) approval for Bylvay (odevixibat) to treat patients aged 12 months and above with cholestatic pruritus caused by Alagille syndrome (ALGS).

FDA Approval 130

FDA Approval Genetics Trials Medicine 130

STAT News

MARCH 11, 2023

The Food and Drug Administration on Friday approved the first treatment for Rett syndrome, a genetic disease mostly affecting girls that causes severe neurologic impairments, robbing them of the ability to communicate or control muscle movement. The new drug, called Daybue, is made by Acadia Pharmaceuticals.

Genetics 98

Genetics FDA Approval Genetic Disease Clinical Trials 98

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.

FDA Approval 130

FDA Approval Gene Therapy Genetic Disease Gene 130

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.

Genetics 130

Genetics DNA Genome Genomics 130

XTalks

FEBRUARY 19, 2025

It occurs when a genetic error causes the overproduction of a protein called CSF1, which attracts inflammatory cells to the joint, often causing pain, swelling and reduced mobility. In 2024, Deciphera was acquired by ONO Pharmaceutical for $2.4 TGCT is a rare, non-cancerous tumor that develops inside or near joints.

FDA Approval 59

FDA Approval Drugs Trials Clinical Trials 59

Pharmaceutical Technology

AUGUST 18, 2022

The US Food and Drug Administration (FDA) has granted approval for bluebird bio ’s Zynteglo (betibeglogene autotemcel, beti-cel) for the treatment of the underlying genetic cause of beta?thalassemia thalassemia in adult and paediatric patients.

Gene Therapy 246

Gene Therapy Gene Genotype In-Vivo 246

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).

FDA Approval 98

FDA Approval Protein Genetics RNA 98

XTalks

NOVEMBER 26, 2024

BridgeBio has been highly active in the genetic diseases space. Its investigational therapy infigratinib recently received Breakthrough Therapy designation, becoming the first-ever potential treatment for achondroplasia — a genetic condition affecting bone growth — to achieve this milestone.

Genetic Disease 105

Genetic Disease Genetics Protein Clinical Development 105

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.

FDA Approval 111

FDA Approval Gene Therapy Gene Trials 111

XTalks

OCTOBER 16, 2024

Hympavzi’s approval, with its innovative approach to balancing clotting, could offer a modern solution that adds to the global effort to reduce the risk of thrombosis. This is Pfizer’s second FDA-approved treatment for a rare genetic blood disorder this year.

FDA Approval 105

FDA Approval Trials Clinical Trials Clinical Trials 105

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.

FDA Approval 105

FDA Approval Protein RNA Clinical Trials 105

XTalks

MARCH 19, 2025

Elevidys is the first FDA-approved gene therapy for DMD a rare genetic disorder characterized by progressive muscle degeneration. Elevidys aims to address the underlying genetic cause by introducing a functional version of the dystrophin gene, which is essential for muscle function.

Gene Therapy 80

Gene Therapy Gene Genetics Clinical Trials 80

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.

Genetics 98

Genetics Genome Genomics Gene 98

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.

FDA Approval 98

FDA Approval Genetics Trials Gene 98

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.

Gene Therapy 128

Gene Therapy Gene FDA Approval Genetics 128

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.

Life Science 90

Life Science FDA Approval Insulin Drugs 90

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.

FDA Approval 102

FDA Approval Sales Trials Development 102

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.

FDA Approval 94

FDA Approval Genetic Disease Genetics Drugs 94

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.

FDA Approval 97

FDA Approval Life Science Clinical Trials Clinical Trials 97

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.

FDA Approval 87

FDA Approval Genetics 87

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.

Gene Therapy 105

Gene Therapy Gene FDA Approval Genetics 105

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.

FDA Approval 85

FDA Approval Genetics Protein Antibody 85

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.

FDA Approval 98

FDA Approval Research Gene Genetics 98

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.

FDA Approval 72

FDA Approval Genetics Genomics Genome 72

Pharmaceutical Technology

FEBRUARY 13, 2023

Gene therapies are an attractive way of targeting the underlying genetic mutations, but traditional approaches cannot be used while targeting mutations for Dravet Syndrome, forcing researchers to develop new ways. GW Pharmaceuticals’ Epidiolex (cannabidiol), the first-FDA approved CBD treatment , is also a valuable addition, he adds.

Research 264

Research Gene Gene Therapy Genome 264

XTalks

MARCH 21, 2025

Following Neffys FDA approval , ARS Pharma reported $2.3 Iovance Biotherapeutics Iovances Amtagvi (lifileucel) is FDA-approved as the first tumor-infiltrating lymphocyte (TIL)-based therapy for solid tumors. With a 30-month shelf life and rapid absorption, Neffy offers an on-the-go alternative to auto-injectors.

Genome 59

Genome Genomics FDA Approval Trials 59

XTalks

DECEMBER 9, 2024

Programs like the Rare Disease Endpoint Advancement Pilot encourage collaboration with the FDA to establish novel surrogate endpoints. This initiative is particularly impactful for rare diseases and genetic disorders, where traditional endpoints may be infeasible.

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Clinical Trials Clinical Trials Compliance Trials 105