KalVista Pharmaceuticals brings to Chiesi Group the product Ekterly, the first FDA-approved oral drug for acute treatment of swelling attacks from the rare disease hereditary angioedema. Ekterly’s tablet formulation offers a dosing edge compared to injectable HAE medications.

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Intellia Therapeutics’ lonvo-z uses CRISPR to inactivate a gene in the liver to reduce levels of a protein key to the swelling attacks from the rare disease hereditary angioedema. Intellia has filed a regulatory submission for this genetic medicine, which could become the first in vivo gene-editing therapy to land FDA approval.

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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.

The post Pharvaris Drug for Rare Swelling Disease Meets Phase 3 Goals; FDA Filing Planned for 2026 appeared first on MedCity News.

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.

The post BioCryst’s $700M Astria Acquisition Gives It a Shot to Beat Larger Rivals in Rare Disease HAE appeared first on MedCity News.

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.

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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.

The post KalVista Drug Gets FDA Approval for Treating Acute Swelling Attacks From Rare Disease appeared first on MedCity News.

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.

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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.

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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.

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