Pharvaris’s deucrictibant met the main and secondary goals of its Phase 3 test as an on-demand treatment for swelling attacks caused by the rare disease hereditary angioedema. Pharvaris’s capsule could compete against Kalvista Pharmaceuticals’ Ekterly, an HAE pill approved by the FDA over the summer.
BioCryst Pharmaceuticals’ Astria Therapeutics acquisition brings navenibart, a drug in Phase 3 testing for the rare disease hereditary angioedema. The injectable antibody has the potential to match the efficacy of the blockbuster Takeda Pharmaceutical drug that currently dominates the market, but with less burdensome dosing that could encourage HAE patients to switch.
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The FDA approved Ionis Pharmaceuticals’ Dawnzera for preventing swelling attacks caused by the rare disease hereditary angioedema. A Takeda Pharmaceutical product dominates this market, but Ionis has clinical data showing patients had better outcomes after switching to Dawnzera from Takeda’s drug and other currently available HAE medications.
KalVista Pharmaceuticals’ Ekterly is now FDA approved for treating acute swelling attacks from hereditary angioedema. The KalVista tablet provides an alternative to injectable or infused HAE medications from companies such as Takeda Pharmaceutical and CSL Behring.
Neopharmed Gentili is acquiring European rights to Orladeyo, bringing the Italy-based pharmaceutical company into rare disease. BioCryst’s Orladeyo is approved for treating patients with the rare disease hereditary angioedema.
In its Phase 3 test, Ionis Pharmaceuticals drug donidalorsen reduced the frequency of swelling attacks caused by the rare disease hereditary angioedema. If Ionis can commercialize this drug, competition would include products from Takeda Pharmaceutical and BioCryst Pharmaceuticals.
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Intellia Therapeutics has encouraging early clinical data for a gene-editing therapy addressing the rare swelling disorder hereditary angioedema. This therapy, the biotech’s second CRISPR-editing based treatment that performs in vivo edits, offers potential for a one-time treatment of a disease that is currently addressed by chronic oral and injectable drugs.
One year after losing Merck as a partner, KalVista reports mid-stage clinical data for a pill that could offer an alternative to injectable drugs for a rare, potentially life-threatening disease. KalVista plans to meet with the FDA about proceeding to a Phase 3 study.