Are haemophilia gene therapies truly “one and done”?
The value of haemophilia gene therapies like Roctavian and Hemgenix hinge on long-term efficacy and innovative payment structures play a key role.
21 November 2023
21 November 2023
The value of haemophilia gene therapies like Roctavian and Hemgenix hinge on long-term efficacy and innovative payment structures play a key role.
Oxurion’s diabetic macular oedema drug failed to meet its primary endpoint in the Phase IIb trial.
The lawsuit claims that Pfizer misrepresented that the ADHD drug was compliant with the law, hiding the fact that it was adulterated.
NeoImmuneTech is developing NT-17, an IL-7 treatment, to promote rapid T-cell recovery in patients with acute radiation syndrome.
Zevra has obtained access to Acer's rare disease assets Olpruva and Edsivo.
MIRALOGX will provide a $3m line of credit to finance Ketamir-2’s initial development.
Takhzyro has been approved for the routine prevention of recurrent hereditary angioedema attacks in patients aged two years and older.
The submission of sBLA is supported by findings from the Phase III MARIPOSA-2 clinical trial of Rybrevant.
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Only a handful of approved therapeutic drugs are currently available for the treatment of ARF, all belonging to the solute carrier family 12 member 1 inhibitor. The clinical trial space in ARF consists of almost an equal mix of commercial as well as academic sponsors, with Iran and the US emerging as the key countries for conducting Phase III trials. While the current marketed drug space for ARF has only a handful of treatment options, currently available mid-to-late-stage pipeline drugs are likely to pave the way for a new treatment approach in the future.
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