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Delivery systems for biologics

European Pharmaceutical Review

1 However, the entry of biologics into the pharmaceutical market is not without unique challenges, particularly when compared to small molecule drugs. Indeed, in 2022, biologics constituted 40 percent of all US Food and Drug Administration (FDA) approved drugs, projecting a compound annual growth rate (CAGR) of 9.5

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Ocugen’s retinal therapy gains FDA orphan drug status

Pharmaceutical Technology

Ocugen has received orphan drug designation from the US Food and Drug Administration for its OCU410ST (AAV5-hRORA) to treat ABCA4 – linked retinopathies. The gene mediates the transport and removal of all-trans-retinal aldehyde, a byproduct of the retinoid cycle of vision, from photoreceptor cells.

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Lexicon gets FDA approval for Inpefa to treat heart failure

Pharmaceutical Technology

Lexicon Pharmaceuticals (Lexicon) has received approval from the US Food and Drug Administration (FDA) for its Inpefa drug to treat heart failure. Inpefa is a once-daily oral tablet indicated as an inhibitor of sodium-glucose co-transporter type 2 (SGLT2) and type 1 (SGLT1).

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Ipsen concludes purchase of Albireo Pharma

Pharmaceutical Technology

Albireo’s pipeline includes its lead asset, Bylvay (odevixibat), a potent, once-a-day, oral, non-systemic, ileal bile acid transport inhibitor (IBATi). Last month, the US Food and Drug Administration (FDA) accepted Bylvay for Priority Review to treat Alagille syndrome (ALGS) in paediatric and adult patients.

Pharma 52
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Orphalan launches Wilson disease therapy in US

Pharmaceutical Technology

Orphan drug development company Orphalan has introduced Cuvrior, a new trientine tetrahydrochloride (TETA-4HCl), for commercial use in the US for Wilson disease. Wilson disease is a rare inherited disorder of copper transportation in the body and chiefly affects the liver and brain.

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FDA approves Ipsen’s Bylvay for cholestatic pruritus due to ALGS

Pharmaceutical Technology

Ipsen has received US Food and Drug Administration (FDA) approval for Bylvay (odevixibat) to treat patients aged 12 months and above with cholestatic pruritus caused by Alagille syndrome (ALGS).

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mRNA therapeutics: a limitless revolution in medicine

European Pharmaceutical Review

The success of mRNA vaccines against SARS-CoV-2 has quickly catapulted mRNA therapeutics as a disruptive, expanding drug category” The term ‘mRNA’ has become commonplace globally. The success of mRNA vaccines against SARS-CoV-2 has quickly catapulted mRNA therapeutics as a disruptive, expanding drug category.

Medicine 109