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

XTalks

This progressive disease occurs when misfolded transthyretin (TTR) proteins form amyloid deposits in the heart, leading to cardiac dysfunction. Attruby offers a more potent option , following Pfizer’s tafamidis (Vyndaqel and Vyndamax), the first therapy and TTR stabilizer approved to treat ATTR-CM.

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Italfarmaco’s Duvyzat Wins FDA Approval as First Nonsteroidal Treatment for All Genetic Variants of DMD

XTalks

Italy-based drugmaker Italfarmaco has won US Food and Drug Administration (FDA) approval for its oral medication Duvyzat (givinostat) for the treatment of Duchenne muscular dystrophy (DMD) in patients six years of age and older. Duvyzat received Priority Review, Orphan Drug and Rare Pediatric Disease designations from the FDA.

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Roche takes on pricey rivals as FDA approves SMA drug

pharmaphorum

Roche is hoping to undercut hugely expensive rivals after the FDA approved its oral spinal muscular atrophy (SMA) drug Evrysdi (risdiplam). The FDA approved Evrysdi for the treatment of spinal muscular atrophy (SMA) in adults and children two months of age and older.

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Handover of senior R&D roles at Merck continues, as Roy Baynes exits

pharmaphorum

Merck & Co’s chief medical officer and head of clinical development Dr Roy Baynes has announced his retirement from the company to take up a new role alongside his former colleague Roger Perlmutter at biotech Eikon Therapeutics. billion last year.

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First UK patient joins Pfizer’s phase 3 DMD gene therapy trial

pharmaphorum

There are already several drugs on the market to treat Duchenne, notably from Sarepta Therapeutics which has three FDA-approved drugs that use “exon skipping” technology. These allow the body to produce a correct version of the misfolded dystrophin protein that is the root cause of DMD.

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Novavax Announces COVID-19 Vaccine Clinical Development Progress

The Pharma Data

(Nasdaq: NVAX), a late-stage biotechnology company developing next-generation vaccines for serious infectious diseases, today provided an update on its COVID-19 vaccine program. About NVX-CoV2373 NVX-CoV2373 is a protein-based vaccine candidate engineered from the genetic sequence of SARS-CoV-2, the virus that causes COVID-19 disease.

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Look out GSK, Pfizer and BioNTech are coming for Shingrix

pharmaphorum

Having already brought their mRNA-based CVID-19 vaccine Comirnaty to market at breakneck speed, Pfizer and BioNTech are hoping to fast-track a vaccine for shingles based on the same technology platform through clinical development, with trials due to start later this year.