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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pharmaphorum

NOVEMBER 27, 2020

The new EU approval comes after the FDA approved the expanded label last year, and provides the first treatment option that tackles the underlying cause of CF in patients aged six to 11 with these mutations. It is caused by a defective or missing CFTR gene, and there are around 75,000 people worldwide with the disease.

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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.” Takeda’s application for Adzynma was granted a rare pediatric disease priority review voucher, priority review, fast track and orphan designations.

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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.

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FDA Approval Genetic Disease Genetics Clinical Trials 52

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).

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Gene Therapy Gene FDA Approval Dermatology 97

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).

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The Pharma Data

NOVEMBER 24, 2020

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1. Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

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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).

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The Pharma Data

DECEMBER 3, 2020

professor of medicine and clinical director, U.S. In October, the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) granted ORLADEYO a positive scientific opinion through the Early Access to Medicines Scheme (EAMS). The most frequently reported adverse reactions were gastrointestinal related.

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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

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.

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FDA Approval Protein Containment Genetic Disease 64

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.

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FDA Approval Clinical Trials Clinical Trials Dermatology 52

pharmaphorum

JANUARY 10, 2022

With the two approvals acting as a validation of the technology, larger companies are prepared to make larger investments to ensure that they are at the forefront of developments in RNA-focused therapies. Where next?

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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.

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The Pharma Data

NOVEMBER 29, 2020

The FDA approved the drug on November 24. PH1 is an ultra-rare genetic disease marked by overproduction of oxalate, which causes deposits of calcium oxalate crystals in the kidneys and urinary tract and can lead to painful and recurrent kidney stones, nephrocalcinosis, progression to kidney failure, and system organ dysfunction.

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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.

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The Pharma Data

DECEMBER 20, 2020

Elizabeth’s Medical Center and associate professor of Urology at Tufts University School of Medicine, said at the time, “The results of the EMPOUR study over the 52-week period demonstrated the sustained benefits of fibegron. The drug is a once-daily, beta-3 adrenergic agonist. It results in patches of thick, scaly or crusty skin.

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XTalks

DECEMBER 22, 2021

This included Eli Lilly’s partnership with Netherlands-based RNA biotech ProQR Therapeutics this year to develop RNA editing-based therapies for rare genetic diseases, namely blindness-causing retinal conditions, based on the company’s proprietary Axiomer RNA editing platform technology. The deal is worth $1.5

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The Pharma Data

DECEMBER 8, 2020

The assumption is SciNeuro will start with Parkinson’s disease and pain in addition to rare genetic diseases, after scaling up staffing. . A supporting pipeline has medicines to target these key pathways to reverse or eradicate disease and extend the years of healthy life remaining.

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XTalks

SEPTEMBER 25, 2024

Despite extensive research efforts, there have not been approved treatments to meet the significant needs of patients,” said Janet Maynard, MD, MHS, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine (ORPURM), in the FDA’s Center for Drug Evaluation and Research (CDER).

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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.

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Life Science Gene Therapy Gene Manufacturing 52

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. This intersection will be remembered as one of the most significant achievements in science and medicine”. Lipid nanoparticles are spherical vehicles made from ionizable lipids.

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XTalks

JANUARY 6, 2025

Whether its the integration of nanotechnology in medicine, the evolution of point-of-care (POC) diagnostics or the transformative impact of CRISPR and regenerative medicine, these biotech trends are pushing scientific boundaries and creating new opportunities for businesses and researchers alike. billion by 2032, with a CAGR of 10.1

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