Rare Diseases

Pharma News and Updates for Actinium, Apellis, Chiesi, IVERIC, Travere, Takeda

Notizia - Recent Pharma, Healthcare and Biotech Happenings

Actinium Announces Phase III SIERRA Trial Results; FDA Approves Apellis’s Geographic Atrophy Drug; FDA Gives Green Light to Chiesi’s Lamzede; FDA Accepts NDA and Grants Priority Review for Avacincaptad Pegol; FDA Approves Travere’s Kidney Disorder Drug; Takeda Presents Phase 3 GRAPHITE Study Results

Actinium Announces Positive Full Data Results From the Pivotal Phase III SIERRA Trial Actinium Pharmaceuticals, Inc., a pioneer in the development of targeted radiotherapies, announced positive primary and secondary endpoint results from its pivotal Phase III SIERRA trial of Iomab-B in patients aged 55 and older with active relapsed or refractory acute myeloid leukemia (r/r AML). Iomab-B achieved a high level of statistical significance (p0.0001) in the primary endpoint of durable Complete Remission (dCR) six months after initial complete remission following BMT. Iomab-B also improved the secondary endpoint of Event-Free Survival (EFS), with a 78% reduction in the ...

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Biogen’s Aduhelm; FDA Approves Sanofi’s Enjaymo; NHS & Orchard Signs a Deal; Bristol’s Breyanzi & Abecma; Pharming’s Leniolisib; Eli Lilly’s Verzenio Trial; UCB’ Zilucoplan Trials Result; Eli Lilly’s Alzheimer’s Drug Donanemab

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Merck’s Gefapixant; Pfizer’s Somatrogon; Gilead’s Viklury; AbbVie’s Skyrizi; Gilead’s Zydelig Approvals; Lantern’s LP-184; Polpharma to Acquire Advent

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Boston Scientific’s MODULAR CRM System; Restore Medical’s ContraBand clinical trial result; BD acquires Venclose; Roche differentiates mutations in Omicron; Linus’s StrandDx-ASD; HelioLiver launches liquid biopsy test

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Aslan Pharma – IQVIA collaboration; Reata’s kidney drug bardoxolone; Alzheimer’s Disease treatment update; Recordati to acquire EUSA

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Bluebird bio may get gene therapy approved in the US; Neurocrine pens $2.6B pact with Sosei Heptares; FDA opens clearances for hepatitis C diagnostics; Twist nabs antibody discovery biotech Abveris for $190M

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Pfizer tightens DMD trial criteria over safety concerns; Kytopen raises $30M; Eli Lilly taps Reify’s Care Access; Vensana Capital raises $325M

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Exploring the Market Potential of the Upcoming Therapies for the Myasthenia Gravis

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Biogen’s AMD Biosimilar Gets Approval; SmartLabs Series B Funding; BARDA, Partner Therapeutics in Sepsis Space; Synlogic to Explore Sepsis Market Domain

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ADARx Raises $75M Series B; Fast Track Designation for Vor Biopharma’s drug; Ixaka’s cell therapy clears phase 3; Breakthrough Device Designation to NovoTTF-200T™ System

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Rare Diseases

Most Promising Applications of Artificial Intelligence (AI) in Healthcare Segment

Biogen’s Aduhelm; FDA Approves Sanofi’s Enjaymo; NHS & Orchard Signs a Deal; Bristol’s Breyanzi & Abecma; Pharming’s Leniolisib; Eli Lilly’s Verzenio Trial; UCB’ Zilucoplan Trials Result; Eli Lilly’s Alzheimer’s Drug Donanemab

Merck’s Gefapixant; Pfizer’s Somatrogon; Gilead’s Viklury; AbbVie’s Skyrizi; Gilead’s Zydelig Approvals; Lantern’s LP-184; Polpharma to Acquire Advent

The Evolving Neurofibromatosis Treatment Landscape

Boston Scientific’s MODULAR CRM System; Restore Medical’s ContraBand clinical trial result; BD acquires Venclose; Roche differentiates mutations in Omicron; Linus’s StrandDx-ASD; HelioLiver launches liquid biopsy test

Aslan Pharma – IQVIA collaboration; Reata’s kidney drug bardoxolone; Alzheimer’s Disease treatment update; Recordati to acquire EUSA

Bluebird bio may get gene therapy approved in the US; Neurocrine pens $2.6B pact with Sosei Heptares; FDA opens clearances for hepatitis C diagnostics; Twist nabs antibody discovery biotech Abveris for $190M

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Biogen’s ALS drug Tofersen (BIIB067); Phathom’s erosive esophagitis drug trial; Vertex’s islet cell therapy; Natera launches lung DNA test

Pfizer tightens DMD trial criteria over safety concerns; Kytopen raises $30M; Eli Lilly taps Reify’s Care Access; Vensana Capital raises $325M

Exploring the Market Potential of the Upcoming Therapies for the Myasthenia Gravis

Biogen’s AMD Biosimilar Gets Approval; SmartLabs Series B Funding; BARDA, Partner Therapeutics in Sepsis Space; Synlogic to Explore Sepsis Market Domain

ADARx Raises $75M Series B; Fast Track Designation for Vor Biopharma’s drug; Ixaka’s cell therapy clears phase 3; Breakthrough Device Designation to NovoTTF-200T™ System

Which Pipeline Therapy is Expected to Capture the Eyeballs in the Phenylketonuria Market?

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