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FDA Watch: Approvals for Amicus, Alnylam; designations for BioCryst, Neuralstem


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Global M&A By the Numbers: Q1 2022

FDA Watch: Approvals for Amicus, Alnylam; designations for BioCryst, Neuralstem

Approvals and designations made by the U.S. Food and Drug Administration for the week ended Aug. 10.


* Amicus Therapeutics Inc.'s Galafold, for adults with Fabry disease who have a mutation in the galactosidase alpha gene determined to be responsive to treatment. Galafold is already approved in the EU, Japan, Australia, Canada, Israel, South Korea and Switzerland.

* Alnylam Pharmaceuticals Inc.'s Onpattro, for adults with polyneuropathy due to hereditary transthyretin-mediated amyloidosis. Onpattro previously received the FDA's fast track, priority review, breakthrough therapy and orphan drug designations.

* The Population Council Inc.'s Annovera, a reusable combination hormonal contraceptive ring. The nonprofit research organization has a license agreement with TherapeuticsMD Inc. to make Annovera available to women in the U.S.

* Kyowa Hakko Kirin Co. Ltd.'s Poteligeo, for adults with mycosis fungoides and Sézary syndrome whose cancer has returned, or is resistant or nonresponsive to at least one prior therapy. Poteligeo previously received the FDA's breakthrough therapy and orphan drug designations.

* Vertex Pharmaceuticals Inc.'s Orkambi, for children 2 to 5 years old with cystic fibrosis who have two copies of the F508del-CFTR gene mutation. Orkambi is already approved to treat cystic fibrosis in patients ages 6 and older who have two copies of the F508del-CFTR gene mutation.

* Strata Skin Sciences Inc.'s Multi-Micro Dose tip accessory, used to measure and deliver dosage through the company's Xtrac laser system, which treats a variety of skin diseases.

SNL Image
FDA headquarters in Silver Spring, Md.
Source: Associated Press

Complete response letters

* BTG plc's Elevair Endobronchial Coil System, which uses a procedure in which metal coils are placed into the lungs of patients with severe emphysema. BTG said it is reviewing its options.

* Pain Therapeutics Inc.'s Remoxy, a pain drug which is an extended-release oral formulation of the opioid oxycodone. The FDA said the benefits of Remoxy do not outweigh its risks.

Fast track

* BioCryst Pharmaceuticals Inc.'s BCX7353, to prevent and treat swelling attacks in patients with hereditary angioedema.

Breakthrough therapy

* Array BioPharma Inc. combination of Braftovi, Mektovi and cetuximab, for metastatic colorectal cancer. Eli Lilly and Co. markets cetuximab as Erbitux.

Other designations

* Orphan drug, for Neuralstem Inc.'s NSI-189, to treat Angelman syndrome.

* Orphan drug, for Krystal Biotech Inc.'s KB105, to treat transglutaminase 1 deficient autosomal recessive congenital ichthyosis.

* Competitive generic therapy, for Apotex Inc.'s potassium chloride oral solution, to treat low potassium levels in certain patients.

* Rare pediatric disease, for ProMetic Life Sciences Inc.'s PBI-4050, to treat Alström syndrome. PBI-4050 previously received the FDA's orphan drug designation.