rare diseases
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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.
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MedCity Influencers, BioPharma
The Inflation Reduction Act’s Impact on Drug Development for Rare Diseases
Despite these wins for many patients, the new law is already impacting the discovery and development of new drugs for people living with orphan diseases. Not only are drugs that could treat more than one disease being disincentivized, small molecule medicines, which play an important role in treating neurological disorders, cancers, and other diseases, may also be disadvantaged by the law.
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Payer’s Place: Dawn Maroney
Dawn Maroney, President, Markets of Alignment Health and CEO of Alignment Health Plan, to discuss how they are using technology to provide better service and care to consumers.
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Alltrna Unveils $109M to Make tRNA a Universal Therapy for Genetic Diseases
Flagship Pioneering-founded Alltrna is developing tRNA therapies to address genetic diseases driven by truncated versions of proteins. By addressing an aberrant genetic instruction underpinning many diseases, the startup is developing what it describes as a potential “universal solution.”
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MedCity Influencers, BioPharma
The New Opportunity to Help Rare Disease Patients
In 2020, 46.9% of all novel drugs approved by the FDA were for rare diseases, as compared to 23.5% in 2012. Investment is expected to continue trending upward, with estimates suggesting that global spending on rare disease therapies will reach $260 billion by the end of 2025.
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PTC Posts Positive Phase 3 Data, But Comparison to BioMarin Is Less Clear
PTC Therapeutics plans to talk to regulators about regulatory submissions for sepiapterin after the drug met the main goal of a Phase 3 study. The small molecule is a potential treatment for phenylketonuria, a rare enzyme deficiency.
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Startup Nido Bio Reveals $109M (so far) for Precision Approach to Neuro Disorders
Nido Biosciences’ lead drug hits a previously undrugged pocket as a way to potentially treat the rare neuromuscular disorder spinal and bulbar muscular atrophy. The startup’s tech platform screens neurons to find targets for new neurological disorder drugs.
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Travere Therapeutics Drug for Rare Kidney Disease Falls Short in Pivotal Test
Sparsentan failed to meet the main goal of a Phase 3 test in focal segmental glomerulosclerosis (FSGS). Nevertheless, Travere Therapeutics says the totality of data points to patient improvement in the rare kidney disorder and the biotech will talk with regulators about a potential path forward.
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FDA Approval Makes Pharming Drug First for Rare Inherited Immune Disease
Pharming Group drug Joenja was awarded FDA approval for treating a primary immunodeficiency called activated phosphoinositide 3-kinase delta syndrome, or APDS. It’s the first treatment for the rare disorder.
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Reata Pharma Drug Wins First FDA Nod in Ultra-Rare Neuromuscular Disorder
Skyclarys, a Reata Pharmaceuticals drug, is now approved for treating the rare neuromuscular disorder Friedreich’s ataxia. The decision makes the capsule the first therapy for a disease that’s rarer than either Duchenne muscular dystrophy or spinal muscular atrophy.
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Applying Remote Patient Monitoring to Surgery Prep and Recovery, Oncology and Women’s Health
Join us to learn about the latest trends in remote monitoring and how to extend its benefits beyond chronic conditions to more patients – all while using fewer staff resources.
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Chiesi Wins FDA Approval for First Drug to Treat Ultra-Rare Enzyme Deficiency
Chiesi drug Lamzede is an engineered version of an enzyme that’s lacking in patients with the rare disease alpha-mannosidosis. FDA approval of the Chiesi drug comes about five years after European regulators authorized the product.
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Roche Drug for Rare Blood Disorder Hits Phase 3 Goals; Regulatory Filings Are Next
AstraZeneca dominates the market for drugs that treat rare blood disorder paroxysmal nocturnal hemoglobinuria. Roche drug crovalimab met the two main goals of its pivotal test, and if approved, would offer a dosing edge compared to AstraZeneca’s two blockbuster PNH medicines.
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J&J Drug for Rare Infant Blood Disorder Achieves Main Goal of Phase 2 Test
A Johnson & Johnson drug acquired as part of a $6.5 billion deal has preliminary data showing it met the main goal of a mid-stage test in a rare blood disorder that can become fatal to fetuses. Full data from the trial will be presented at a future scientific meeting.
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FDA Asks Gene Therapy Biotech to Run Clinical Trial That Might Be Impossible
Taysha Gene Therapies has encouraging data from an open-label, Phase 1/2 study in the rare disease giant axonal neuropathy. It might not be enough. The FDA recommended the biotech conduct a randomized and placebo-controlled study—a challenge in any ultra-rare disease.
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Artificial Intelligence, BioPharma
Nvidia’s AI Tech Designs Proteins Never Seen in Nature, Pointing Way to New Therapies
A collaboration between Nvidia and startup Evozyne was able to produce novel versions of a human protein never before seen in nature but with enhanced function and the same safety as native protein. The research lays the groundwork for potential new therapies for a rare inherited disorder.
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Sanofi Takes Baton From Kymera to Bring Protein-Degrading Drug Into Phase 2
While most targeted protein degradation drug research has focused on cancer, Kymera Therapeutics’ Phase 1 data bring validation for this approach in treating inflammation. Kymera partner Sanofi has elected to advance the biotech’s program to mid-stage testing in two inflammatory skin disorders.
Promoted
Payer’s Place: Dr. Anil Singh
Dr. Anil Singh shares his insights into the strategies employed by the organization to identify the most effective digital solutions for their members.
Promoted
Discover the Next-Gen Platform for Integrated Collaborative Care
Beyond EHRs and digital front doors, reducing the gaps in patient care journeys.
