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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Delveinsight

AUGUST 17, 2021

FDA’s Green Flag to Keytruda and Lenvima combination by Merck and Eisai for Advanced renal cell carcinoma (RCC). The FDA approved the combination of Keytruda and Lenvima produced by Merck and Eisai, as a first-line treatment of adult patients with advanced renal cell carcinoma (RCC). The rarity of disease – 1.36

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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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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 3, 2020

Food and Drug Administration (FDA) has approved ORLADEYO (berotralstat) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and young patients 12 years and older. The approval was based on data from the Phase 3 APeX-2 trial, which showed that ORLADEYO could significantly reduce attacks at the 24-week mark.

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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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Pharmaceutical Technology

JULY 29, 2022

CAMP4’s CSO David Bumcrot PhD tells Pharmaceutical Technology that the company plans to see clinical trials go forward for their urea cycle disorder programs late next year. Ultragenyx Pharmaceuticals has an orthinine transcarbamylase (OTC) activator drug in Phase III trials.

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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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XTalks

FEBRUARY 27, 2023

Chiesi Global Rare Diseases recently announced in a press release that Lamzede (velmanase alfa-tycv) received US Food and Drug Administration (FDA) approval for the treatment of non-neurological manifestations of alpha-mannosidosis in both adult and pediatric patients.

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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. Verifying the biomarker’s clinical validity for use as a surrogate endpoint in rare disease research is another hurdle which is generally a longer-term goal.

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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.

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pharmaphorum

SEPTEMBER 28, 2022

Clinical trial results reported at this year’s European Haematology Association (EHA) congress showed that a one-time treatment with exa-cel had a significant benefit in both SCD and thalassaemia patients. .

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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?

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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. Advisors also had some concerns about the safety of the drug and the design of the pivotal trial used to support the marketing application.

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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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Drug Discovery World

NOVEMBER 14, 2023

Adzynma provides patients with a treatment option that replaces their deficient ADAMTS13 enzyme and offers a favourable efficacy and safety profile and reduced administration time and volume compared to current plasma-based therapies.

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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. Nulibry Trial Results.

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The Pharma Data

NOVEMBER 29, 2020

The submission was based on the safety and efficacy data of the pivotal Phase II trials 201 and 12-230. The FDA approved the drug on November 24. It works by degrading HAO1 mRNA and decreasing the synthesis of glycolate oxidase (GO), an enzyme upstream of the disease-causing defect in PH1.

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The Pharma Data

DECEMBER 20, 2020

It was approved on December 15, 2020. The submission included data from two positive pivotal Phase III trials. The FDA approval of Klisyri is a significant milestone for Athenex,” said Johnson Lau, chairman and chief executive officer of Athenex. and Europe, including Russia. Most Read Today. Source link.

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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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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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The Pharma Data

DECEMBER 8, 2020

The ImmunoTAC platform pairs proprietary payloads that modulate key disease modifying pathways with monoclonal antibodies directed at specific disease sites. Silverback’s lead candidate is currently in a Phase I trial in adults with HER2-expressing solid tumors. Nuance Pharma .

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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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Drug Discovery World

MARCH 19, 2024

Lenmeldy (atidarsagene autotemcel) has become the first Food and Drug Administration (FDA)-approved gene therapy 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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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.

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XTalks

DECEMBER 22, 2021

We talked about the emergence of dozens of CRISPR biotechs last year and the growing number of CRISPR-based therapies in clinical trials for conditions ranging from Duchenne muscular dystrophy (DMD) and sickle cell disease (SCD). The deal is worth $1.5 Prior to this, Lilly struck an agreement worth $1.25

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Drug Discovery World

FEBRUARY 22, 2024

For example, it can take up to it three years to recruit enough participants for a dementia clinical trial. years it takes to complete an entire cancer clinical trial. The organisation also notes that only 1% of the people that can take part in dementia clinical trials actually do so. This compares to the 2.3

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The Pharma Data

NOVEMBER 25, 2020

The mRNA vaccine candidate developed by Pfizer and Germany-based BioNTech demonstrated 95% efficacy in clinical trials. Food and Drug Administration (FDA) will review the data on Dec. The two genetic diseases cause premature, rapid aging that dramatically decreases the lifespan of children affected.

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