Zevra Therapeutics’ Miplyffa (arimoclomol) has received FDA approval for treating neurological symptoms associated with Neimann-Pick disease type C (NPC) in adults and children. This is the first drug approved for this rare genetic disease, offering hope to patients suffering from its progressive neurological symptoms and organ dysfunction.
Stifel has initiated coverage on Inozyme Pharma (INZY), highlighting the potential of its lead candidate, INZ-701, an enzyme replacement therapy (ERT) for rare diseases. The analyst believes the drug has pipeline-in-a-product potential and forecasts a Buy rating with a price target of $16.
The Indian government has included primary hyperoxaluria, a life-threatening kidney, bladder, and urinary tract condition, under its National Policy for Rare Diseases, providing financial assistance of up to ₹ 50 lakh for patients requiring kidney transplants. This inclusion signifies the government’s commitment to supporting individuals battling rare diseases and ensuring access to life-saving treatments.
The global acute intermittent porphyria (AIP) market is projected to experience substantial growth, driven by advancements in AI, digital health technologies, and patient-centric drug development. AI-powered tools are enabling personalized treatment strategies and improved outcomes, while telemedicine and remote monitoring are revolutionizing AIP care delivery. This market analysis explores key trends, regional profiles, and prominent players shaping the future of AIP treatment.
Ascendis Pharma’s Yorvipath (palopegteriparatide) has received FDA approval for the treatment of hypoparathyroidism in adults. This approval fills a crucial gap in treatment options for this rare condition, particularly as Takeda’s Natpar/Natpara will be discontinued in 2024. Yorvipath is expected to be available in the first quarter of 2025.
Incyte (NASDAQ: INCY) has entered into an agreement to acquire Escient Pharmaceuticals, a privately held biotechnology company focused on developing novel therapeutics for immune and neuro-immune disorders. The transaction, valued at $750 million plus Escient’s net cash, is expected to close in the third quarter of 2024, subject to customary closing conditions. Upon completion, Incyte will gain exclusive rights to Escient’s portfolio of compounds, including EP262 and EP547, which target the MRGPRX2 receptor expressed on mast cells. EP262 is currently being evaluated in a mid-stage clinical trial for chronic spontaneous urticaria, a type of allergic skin condition. The acquisition is expected to strengthen Incyte’s position in the inflammatory disease space and provide potential launch opportunities starting in 2029.
Gene therapy, a groundbreaking medical advancement, offers immense promise for treating genetic disorders. However, the absence of long-term safety data poses challenges. Early evidence-development strategies and cautious early coverage decisions are crucial to ensure the safest and most beneficial outcomes for patients and society.
KKR, a global investment firm, has acquired Immedica Pharma, a Stockholm-based pharmaceutical company specializing in rare diseases and specialty care products. The acquisition will involve the reinvestment of Immedica’s existing owner, Impilo, which will become an equal owner alongside KKR. The partnership aims to support Immedica’s continued growth, including the launch of its recently approved ultra-orphan drug Loargys, additional pipeline assets, and continued in-licensing and mergers and acquisitions.
Tracy Dixon-Salazar’s daughter, Savannah, was diagnosed with epilepsy as a child, but her seizures became severe and frequent. Faced with limited answers from doctors, Dixon-Salazar returned to school and earned a Ph.D. in neurobiology. Through her research, she discovered that a calcium channel mutation was causing Savannah’s seizures and found a treatment that reduced them by 95%. Dixon-Salazar now advocates for families affected by Lennox-Gastaut syndrome, highlighting the challenges of caregiving and the importance of support.
CustomerInsights.AI (CIAI), a provider of technology-enabled solutions for Life Sciences companies, has partnered with Ambit, a consulting firm specializing in rare and specialty diseases.
This partnership combines CIAI’s scalable data architecture, automated workflows, and AI capabilities with Ambit’s consulting expertise in rare diseases. Together, they aim to accelerate the development and deployment of data and analytics solutions for rare and specialty disease companies.
