Clinical Trials, FDA Approval, Genetic Disease and Life Science

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FDA Approval of Sanofi’s Enzyme Replacement Drug for ASMD + Disney Actress Partners with Medtronic for Diabetes Tech Campaign – Xtalks Life Science Podcast Ep. 77

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

Life Science FDA Approval Insulin Drugs

Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio

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.

Gene Therapy

Gene Therapy Clinical Trials Clinical Trials Gene

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Trending Sources

4 Life Sciences Trends for 2023

XTalks

DECEMBER 29, 2022

The life sciences and healthcare are among the biggest industries globally, and their significance was particularly highlighted during the past couple of years by the COVID-19 pandemic. Given the hyperfocus on the life sciences thanks to COVID, consumers appear to be more autonomous and vocal about their medical demands and choices.

Life Science

Life Science Gene Therapy Gene Manufacturing

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

XTalks

NOVEMBER 10, 2023

“As we strive to help patients with limited or no treatment options, developing innovative treatments in rare diseases is an inspiring challenge and one we have taken on for 70-plus years as a leader in hematology. No adverse events were reported during the clinical trials following the administration of Adzynma.

FDA Approval

FDA Approval Gene Therapy Gene Clinical Trials

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

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.”

Gene Therapy

Gene Therapy FDA Approval Gene In-Vivo

Amicus’ Pombiliti and Opfolda Is a New Therapy for Pompe Disease

XTalks

NOVEMBER 27, 2023

Amicus Therapeutics has recently received approval from the US Food and Drug Administration (FDA) for its drugs Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) for adults living with late-onset Pompe disease (LOPD) who are not improving on their current enzyme replacement therapy (ERT).

FDA Approval

FDA Approval Clinical Trials Clinical Trials Trials

Spevigo Becomes First Treatment Option for Generalized Pustular Psoriasis Flares

XTalks

SEPTEMBER 22, 2022

On September 1, 2022, Boehringer Ingelheim Pharmaceuticals announced in a press release that the US Food and Drug Administration (FDA) approved Spevigo (spesolimab-sbzo) intravenous injections for GPP flares in adults. Spevigo is the first approved treatment option for GPP in adults. Clinical Trial Behind Spevigo.

FDA Approval

FDA Approval Clinical Trials Clinical Trials Dermatology

Sharing Colours on Rare Disease Day 2022

XTalks

FEBRUARY 28, 2022

Related: UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD. Below are some facts and information about rare diseases, including rare disease clinical trials and orphan drugs. How is a Rare Disease Defined? Are Most Rare Diseases Genetic?

Clinical Trials

Clinical Trials Clinical Trials Genetics Trials

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

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

XTalks

MAY 12, 2023

In March, the US Food and Drug Administration (FDA) approved Daybue (trofinetide) , the first and only treatment for Rett syndrome. Daybue (trofinetide) is the first and only FDA-approved drug for Rett syndrome. Dr. Bishop: Rett syndrome is a genetic disorder caused by a mutation in the MECP2 gene.

FDA Approval

FDA Approval Genetics Trials Drugs

Key Trends in the Life Sciences to Look Forward to in 2022

XTalks

DECEMBER 22, 2021

If the past year is anything to go by, then 2022 will also be a year marked by continued innovations in the life sciences. The development and widespread adoption of new technologies is key to revolutionizing the way we diagnose, prevent, treat and manage disease. The RNA Revolution: From mRNA Vaccines to RNA Editing.

Life Science

Life Science RNA Vaccination Vaccine

BridgeBio Pharma’s Nulibry Approved as First Treatment for Molybdenum Cofactor Deficiency Type A

XTalks

MARCH 3, 2021

The approval was granted to BridgeBio Pharma, Inc. BridgeBio is dedicated to developing therapies for genetic diseases with unmet needs. The announcement was made on Rare Disease Day, which raises awareness about the impact of rare diseases on patients and their caregivers. Nulibry Trial Results.

FDA Approval

FDA Approval Genotype Clinical Trials Clinical Trials

Biopharma Money on the Move: December 2 – 8

The Pharma Data

DECEMBER 8, 2020

December is bringing frost in the North and plenty of cold hard cash for these life sciences companies. . European venture capital firm Forbion rounded up $545 million for its fifth life sciences fund. The impact of CNS diseases extends beyond patients—to their families and society as well.”

RNA

RNA Antibody Life Science Protein

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

XTalks

SEPTEMBER 17, 2020

Acromegaly Surrogate Endpoint: Serum growth hormone and serum insulin-like growth factor 1 (IGF-1) are acceptable surrogate endpoints for acromegaly clinical trials involving somatostatin analogs such as octreotide, lanreotide and pasireotide. Novartis’ Signifor (pasireotide) is also indicated in the treatment of the disease.

Trials

Trials Gene Hormones Clinical Trials

Lenmeldy, First Approved MLD Gene Therapy for Children, Becomes Most Expensive Drug in the US at $4.25 Million

XTalks

MARCH 21, 2024

The US Food and Drug Administration (FDA) has awarded approval to Orchard Therapeutics for its gene therapy Lenmeldy (atidarsagene autotemcel) for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).

Gene Therapy

Gene Therapy Gene Drugs FDA Approval

Clinical Research Informer

FDA Approval of Sanofi’s Enzyme Replacement Drug for ASMD + Disney Actress Partners with Medtronic for Diabetes Tech Campaign – Xtalks Life Science Podcast Ep. 77

Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio

Trending Sources

4 Life Sciences Trends for 2023

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

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

Amicus’ Pombiliti and Opfolda Is a New Therapy for Pompe Disease

Spevigo Becomes First Treatment Option for Generalized Pustular Psoriasis Flares

Sharing Colours on Rare Disease Day 2022

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

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

Key Trends in the Life Sciences to Look Forward to in 2022

BridgeBio Pharma’s Nulibry Approved as First Treatment for Molybdenum Cofactor Deficiency Type A

Biopharma Money on the Move: December 2 – 8

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

Lenmeldy, First Approved MLD Gene Therapy for Children, Becomes Most Expensive Drug in the US at $4.25 Million

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