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.
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.
At the Payer Insights sessions on Day 1 of ViVE 2024, a panel on prior authorization offered compelling insights from speakers who shared the positive developments in this area after years of mounting frustration. Speakers also shared challenges as they work with providers to figure out how policy developments and technology will work in practice.
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.
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’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.
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.
Canada has a proud history of achievement in the areas of science and technology, and the field of biomanufacturing and life sciences is no exception.
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.
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.
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.”
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.
This eBook, in collaboration with Care Logistics, details how hospitals and health systems can facilitate more effective decision-making by operationalizing elevated awareness.
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.
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.
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.
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.
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.