FDA Approval, Genetic Disease, Genetics and Regulation

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US FDA approves Krystal Biotech’s Vyjuvek for DEB

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

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Sanofi’s Enzyme Replacement Therapy Xenpozyme Wins FDA Approval for Rare Disease ASMD

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.

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Adzynma Wins FDA Approval as First Treatment for Ultra-Rare Blood Clotting Disorder cTTP

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.

FDA Approval

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FDA Approves Imcivree (setmelanotide) for Chronic Weight Management in Patients with Obesity Due to POMC, PCSK1 or LEPR Deficiency

The Pharma Data

NOVEMBER 27, 2020

FDA Approves Imcivree (setmelanotide) for Chronic Weight Management in Patients with Obesity Due to POMC, PCSK1 or LEPR Deficiency. Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, announced today that the U.S. BOSTON, Nov.

FDA Approval

FDA Approval Genetic Disease Genetics Clinical Trials

Vertex gets EU okay to treat younger cystic fibrosis patients

pharmaphorum

NOVEMBER 27, 2020

A new EMA approval has expanded the use of Vertex Pharma’s exon-skipping cystic fibrosis therapy Symkevi to children as young as six if they have specific gene mutations. Symkevi has been an option for CF patients in this group aged 12 or over for two years, but the new approval means treatment will now be able to begin much earlier.

Gene

Gene Genetic Disease FDA Approval Genetics

After Daybue’s Approval: Acadia Pharmaceuticals’ Next Steps for the Rett Syndrome Community

XTalks

MAY 12, 2023

Rett syndrome is a rare genetic disorder that predominantly affects girls and leads to severe physical and cognitive impairments. The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. Daybue (trofinetide) is the first and only FDA-approved drug for Rett syndrome.

FDA Approval

FDA Approval Genetics Trials Drugs

FDA advisors unconvinced by Reata’s kidney disease drug

pharmaphorum

DECEMBER 9, 2021

An FDA advisory committee has delivered a blow to Reata Pharma, after voting unanimously that the drugmaker’s data on bardoxolone – a drug for kidney disease – did not show it is effective. In severe firms, the disease leads to progressive kidney damage with patients often needing dialysis.

Drugs

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First therapy for very rare blood clotting disorder gets US greenlight

Drug Discovery World

NOVEMBER 14, 2023

A very rare, inherited blood clotting disorder, cTTP is caused by a disease-causing mutation in the ADAMTS13 gene, which is responsible for making an enzyme, also named ADAMTS13, that regulates blood clotting. A deficiency in this enzyme causes blood clots to form in the small blood vessels throughout the body.

FDA Approval

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GW Pharmaceuticals’ Epidiolex Receives Approval for Second Rare Disease Indication

XTalks

AUGUST 6, 2020

The US Food and Drug Administration (FDA) has approved the use of an oral cannabidiol (CBD) solution called Epidiolex for the treatment of seizures associated with tuberous sclerosis complex (TSC) in patients aged one year and older. Epidiolex is the only FDA-approved formulation that contains CBD derived from the cannabis plant.

FDA Approval

FDA Approval Protein Containment Genetic Disease

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

Gene Therapy

Gene Therapy Gene Trials Clinical Trials

4 Life Sciences Trends for 2023

XTalks

DECEMBER 29, 2022

As of December 16, 2022, there are seven US Food and Drug Administration (FDA) approved gene therapies. In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. In 2023, a number of gene therapies are expected to get the FDA green light.

Life Science

Life Science Gene Therapy Gene Manufacturing

Advances in neuroscience drug discovery

Drug Discovery World

FEBRUARY 22, 2024

For diseases as serious and debilitating for patients and their families as leukodystrophies, learning about such advances in knowledge carries a great deal of hope, which IRCM warmly welcomes,” adds Dr Jean-François Côté, the IRCM’s President and Scientific Director.

Drugs

Drugs Gene Clinical Trials Clinical Trials

Surrogate Endpoints: How to Choose the Best One for Your Rare Disease Trial

XTalks

SEPTEMBER 17, 2020

In rare disease trials, it’s not always feasible to choose clinically-relevant endpoints to measure the efficacy of a new therapeutic. Surrogate endpoints were used as the basis for approval of 45 percent of new drugs reviewed by the FDA between 2010 and 2012. Nizoral is prescribed off-label, while the others are FDA approved.

Trials

Trials Gene Hormones Clinical Trials

Covid-19 vaccine success bolsters nanoparticle drug delivery research

Pharmaceutical Technology

APRIL 24, 2023

Covid-19 vaccines from Moderna and Pfizer /BioNTech, which use lipid nanoparticles, became the only two FDA-approved vaccines for almost all ages. However, after a period of slow movement in the field, the FDA approved the first nanodrug, Doxil, for Kaposi sarcoma in 1995.

Drug Delivery

Drug Delivery Vaccination Vaccine Research

Clinical Research Informer

US FDA approves Krystal Biotech’s Vyjuvek for DEB

Sanofi’s Enzyme Replacement Therapy Xenpozyme Wins FDA Approval for Rare Disease ASMD

Trending Sources

Adzynma Wins FDA Approval as First Treatment for Ultra-Rare Blood Clotting Disorder cTTP

FDA Approves Imcivree (setmelanotide) for Chronic Weight Management in Patients with Obesity Due to POMC, PCSK1 or LEPR Deficiency

Vertex gets EU okay to treat younger cystic fibrosis patients

After Daybue’s Approval: Acadia Pharmaceuticals’ Next Steps for the Rett Syndrome Community

FDA advisors unconvinced by Reata’s kidney disease drug

First therapy for very rare blood clotting disorder gets US greenlight

GW Pharmaceuticals’ Epidiolex Receives Approval for Second Rare Disease Indication

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

4 Life Sciences Trends for 2023

Advances in neuroscience drug discovery

Surrogate Endpoints: How to Choose the Best One for Your Rare Disease Trial

Covid-19 vaccine success bolsters nanoparticle drug delivery research

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