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 64

FDA Approval Protein Containment Genetic Disease 64

XTalks

SEPTEMBER 25, 2024

In a significant regulatory milestone, the US Food and Drug Administration (FDA) approved Aqneursa (levacetylleucine) for treating neurological symptoms associated with Niemann-Pick disease type C (NPC). NPC, caused by mutations in the NPC1 or NPC2 gene, typically shortens life expectancy to around 13 years.

FDA Approval 52

FDA Approval Drugs Protein Gene 52

XTalks

MAY 12, 2023

The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. 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.

FDA Approval 97

FDA Approval Genetics Trials Drugs 97

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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RNA Vaccine Vaccination Pharma Companies 111

XTalks

DECEMBER 22, 2021

RNA, and its protein-generating form messenger RNA (mRNA) discovered in 1961 , has quickly transitioned from being an obscure, finicky molecule that is difficult to work with, to becoming a significant cornerstone of therapeutic innovation in pharma and biotech. The RNA Revolution: From mRNA Vaccines to RNA Editing. The deal is worth $1.5

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Life Science RNA Vaccine Vaccination 98

The Pharma Data

DECEMBER 20, 2020

The FDA approval of Klisyri is a significant milestone for Athenex,” said Johnson Lau, chairman and chief executive officer of Athenex. CF is a rare, life-shortening genetic disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. Most Read Today. Source link.

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In-Vitro FDA Approval Drugs Manufacturing 52

The Pharma Data

DECEMBER 8, 2020

Through its Shielded Living Therapeutics platform, the company is developing functional cures for chronic diseases. The impact of CNS diseases extends beyond patients—to their families and society as well.” Pear’s reSET, reSET-O and Somryst are the first PDTs to receive FDA approval for treating disease.

RNA 52

RNA Antibody Life Science Protein 52

XTalks

SEPTEMBER 17, 2020

Janssen’s Nizoral (ketoconazole), Bristol Myers Squibb’s Lysodren (mitotane), Novartis’ Metopirone (metyrapone) and Corcept Therapeutics’ Korlym (mifepristone) are all cortisol-inhibiting medications that may be prescribed to treat Cushing’s disease. Novartis’ Signifor (pasireotide) is also indicated in the treatment of the disease.

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Trials Gene Hormones Clinical Trials 98

XTalks

JANUARY 3, 2025

For the first time in the US, adults living with familial chylomicronemia syndrome (FCS) have an FDA-approved treatment option. Tryngolza works by targeting a protein in the liver, apoC-III, which regulates triglyceride metabolism. FCS also severely impacts quality of life, causing chronic fatigue and recurrent stomach pain.

FDA Approval 59

FDA Approval Drugs Clinical Trials Clinical Trials 59

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.

FDA Approval 52

FDA Approval Life Science Containment Genetics 52

XTalks

JANUARY 6, 2025

Moreover, AI has the potential to identify features and signs of disease that may be overlooked by human experts, offering a new layer of diagnostic capability. Notably, the ASO drug Vitravene (fomivirsen) received US Food and Drug Administration (FDA) approval for treating cytomegalovirus (CMV) retinitis in AIDS patients.

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Gene Therapy RNA Gene Editing Gene 111

XTalks

FEBRUARY 13, 2025

Gomekli is the first treatment approved for both adults and children with the disorder. In 2020, AstraZeneca and Merck won FDA approval for Koselugo (selumetinib) to treat NF1-PNs, but its approval was limited to pediatric patients. Gomekli is SpringWorks’ second FDA approval in the past 15 months.

FDA Approval 59

FDA Approval Genetics Clinical Trials Clinical Trials 59