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bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Pharmaceutical Technology

These approvals represent crucial milestones for bluebird bio, the gene therapy field, and patients with rare genetic diseases. Prior to bluebird's approvals, there were only two FDA-approved gene therapies for inherited conditions on the market.

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The downside of VC funding for biotech

World of DTC Marketing

The amount of money required to gain approval of a new drug has been hotly debated, but it also costs a hell of a lot of money to launch a drug in today’s market. A study in 2020 estimated that the median cost of getting a new drug into the market was $985 million, and the average price was $1.3

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

XTalks

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.”

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CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder. Drugs for dialysis, amino acid supplements, and drugs which convert blood ammonia are marketed right now as the go-to urea cycle disorder treatments.

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FDA approval to Keytruda and Lenvima; FibroGen’s Roxadustat; Pfizer’s TICOVAC; Ipsen’s Palovarotene; Jazz Pharma’s Xywav; Seagen-RemeGen’s Cancer Medicine

Delveinsight

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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BridgeBio’s Attruby (Acoramidis): A New, More Potent TTR Stabilizer for ATTR-CM

XTalks

A Marketing Authorization Application has been submitted to the European Medicines Agency (EMA), with a decision anticipated in 2025. BridgeBio has been highly active in the genetic diseases space. From a regulatory standpoint, BridgeBio has taken steps to ensure global access to Attruby.

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

XTalks

“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. The study evaluated both prophylactic and on-demand Adzynma ERT compared to plasma-based therapies in cTTP patients.