Acute Intermittent Porphyria (AIP) is a rare genetic disorder that affects the production of heme, a component of hemoglobin. While there may not be specific companies exclusively focused on AIP due to its rarity, pharmaceutical companies, research institutions, and healthcare organizations may be involved in the development of treatments, diagnostic tools, and support for individuals with AIP.
Latest Acute Intermittent Porphyria Companies Updates:
Alnylam PharmaceuticalsPositive topline results from Phase 3 ENVISION study of givosiran, an investigational RNAi therapeutic for acute hepatic porphyria (including AIP).Givosiran granted Breakthrough Therapy Designation by the US FDA.Ongoing Phase 3 ENVISION2 study of givosiran for AIP prophylaxis.
Apellis Pharmaceuticals Development of APL-1202, a pegylated hemin product for the treatment of acute porphyria attacks.Phase 2b study of APL-1202 ongoing, with results expected in 2024.
Akcea TherapeuticsMarketing Lumitrio (givosiran) for the treatment of acute hepatic porphyria in the US and Europe.Expanding access to Lumitrio through patient assistance programs and collaborations with healthcare providers.
BioMarin PharmaceuticalMarketing Vyndaqel (tabeleumac) for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR), which can have some overlapping symptoms with AIP.Continuing research and development efforts for other rare diseases, including porphyrias.
List of Acute Intermittent Porphyria Key companies in the market:
Acute Intermittent Porphyria Market Highlights:
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