Why Synthetic Data is the Antidote to Clinical Trials
To address the clinical burden and enhance R&D, companies are turning to virtual solutions. This involves synthetic data, digital twin models, and AI to speed analysis.
Neurocrine’s $2.9B Soleno Buyout Brings What Could Become Its Next Blockbuster Drug
Soleno Therapeutics brings Neurocrine Biosciences Vykat XR, a drug commercialized for treating the rare genetic disease Prader-Willi syndrome. This blockbuster prospect complements Crenessity, a Neurocrine drug approved for treating a rare endocrine disorder called congenital adrenal hyperplasia.
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Survey on the Healthcare Financial Landscape Offers a Roadmap for Stakeholders
Zelis highlights how employers, payers, and consumers are integrating digital tools to manage healthcare costs.
The Lethal Cost of Regulatory Perfection in Rare Disease
Congress has already granted the FDA flexibility in evaluating therapies for rare diseases, including the use of real-world evidence and natural history data when traditional large-scale trials are not feasible. The question before the FDA now is not if those tools can be applied – they can – but if the agency has the courage to use them before more patients lose their autonomy, and ultimately, their lives, to rare disease.
We Have a Problem: Even Doctors Struggle to Navigate our Healthcare System
I lived for nine years with ALS while navigating a healthcare system that routinely obscured my own medical reality. But I know that pain is only amplified for individuals without medical expertise like mine.
How AI Enables Unified Patient Journeys for Faster, More Accurate Rare Disease Diagnoses
Every year shaved off the diagnostic odyssey means fewer irreversible complications, fewer misdiagnoses, and fewer clinicians and families left wondering what they missed. A unified, longitudinal approach doesn’t just speed up diagnosis — it changes, and in some cases saves, lives.
Novo Nordisk Gets New Complement System Approach to Rare Diseases With Deal for Omeros Drug
Novo Nordisk is paying $240 million up front for global rights to zaltenibart, an Omeros drug that blocks the MASP-3 protein to treat diseases caused by excessive activity of the complement system. This antibody drug is on track for pivotal testing that could show superiority over blockbuster AstraZeneca drugs that are standard treatments for a rare blood disorder.
The Hidden Administrative Tasks Draining Small Practices
Small practices play a critical role in healthcare delivery, but they cannot continue to absorb ever-increasing administrative demands without consequences.
Chiesi’s Rare Disease Strategy Expands to Genetic Meds via Alliance With CRISPR Startup Arbor
Chiesi Global Rare Diseases is committing up to $115 million to begin a collaboration on Arbor Biotechnologies’ in vivo gene-editing therapy for primary hyperoxaluria type 1, an inherited liver disorder. It’s the rare disease company’s first foray into genetic medicines.
BioMarin’s $270M Inozyme Acquisition Brings Drug That Could Become First for a Rare Disease
Inozyme Pharma’s main asset is in pivotal testing in children who have a rare enzyme deficiency that affects bones and blood vessels. BioMarin Pharmaceutical, already well established in enzyme replacement, said it will use its experience and infrastructure to grow the market for this product, potentially for several indications.
Gene Therapy Is Now First FDA-Approved Treatment for Rare & Fatal CNS Disorder
Orchard Therapeutics gene therapy Lenmeldy won FDA approval for treating the rare enzyme deficiency metachromatic leukodystrophy. In January, Kyowa Kirin completed its acquisition of Orchard, which now operates as a subsidiary of the Japanese drugmaker.
Genetic Medicines Biotech Dyne Pulls In $345M for Clinical Tests of Muscle Drugs
Dyne Therapeutics recently reported encouraging Phase 1/2 clinical data in myotonic dystrophy type 1 and Duchenne muscular dystrophy. At the J.P. Morgan Healthcare Conference, CEO Joshua Brumm said Dyne’s existing cash combined with the new capital is expected to last through 2025—well beyond the next key milestones for both therapeutic candidates.
The Power Behind Enterprise EHR Software for Large Healthcare Systems
Enterprise EHR boosts scalability, interoperability, and governance for large healthcare systems.
FDA Rejection Is a Delay, Not a Detour for Alnylam’s Aim to Treat Heart Disorder
Despite an affirmative FDA advisory committee vote, the agency declined to approve Alnylam Pharmaceuticals’ Onpattro for treating the heart complications caused by a rare, inherited protein disorder. But Alnylam has other drugs candidates for the disease, including one expected to post Phase 3 data in the first half of 2024.
Kyowa Kirin Buys Into Gene Therapy With Orchard Therapeutics Acquisition
The Orchard Therapeutics acquisition comes as the biotech’s gene therapy for metachromatic leukodystrophy approaches a March 2024 FDA decision. The ex vivo gene therapy is already commercialized in Europe, marketed as Libmeldy.
Novartis Kidney Disease Drug Hits First Goal of Study; 2024 FDA Filing Planned
Novartis’s iptacopan met the first of two main goals in its pivotal study in immunoglobulin A nephropathy, and the pharma giant plans to seek accelerated FDA approval next year. The drug could challenge two available therapies for the rare kidney disorder.
Amicus Therapeutics Rare Disease Combo Therapy Wins Long-Awaited FDA Nod
Sanofi dominates the market for therapies that treat Pompe disease, a rare enzyme deficiency. The FDA has approved an Amicus Therapeutics combination treatment for patients inadequately served by the Sanofi products.
Amgen, FTC Settlement Allows $28B Horizon Acquisition to Move Forward
Amgen and the Federal Trade Commission are settling the lawsuit the regulator filed to block the pharmaceutical giant’s $28 billion Horizon Therapeutics acquisition. As part of the settlement, Amgen agrees not to “bundle” its products with Horizon’s drugs in negotiations with health plans.