AstraZeneca raises 2022 guidance, names Demaré as non-exec chair
AstraZeneca chief executive Pascal Soriot said this morning that strong revenue increases in 2022 – fuelled by its COVID-19 antibody Evusheld and oncology drugs – […]
AstraZeneca chief executive Pascal Soriot said this morning that strong revenue increases in 2022 – fuelled by its COVID-19 antibody Evusheld and oncology drugs – […]
Decipher Complement Pathway Intricacies, Validate Pathway Activation Assays & Discover Next-Generation Complement-Targeted Approaches that are Transforming Patient Outcomes With promising clinical data, an increasing number […]
Novartis is acquiring an FDA priority review voucher from Mallinckrodt Pharmaceuticals. The $100 million price tag is in the neighborhood of the going rate for […]
Sanofi won European Commission approval for two therapies that treat rare enzyme deficiencies. Xenpoyzme is the first treatment for acid sphingomyelinase deficiency (ASMD) while Nexviadyme […]
R&D into orphan drugs is growing alongside the number of approved treatments, providing treatments for rare diseases that previously did not have any. However, Ben […]
Roche drug Evrysdi, one of three FDA-approved treatments for spinal muscular atrophy, is now approved for treating those younger than age 2. The regulatory decision […]
Biohaven’s migraine drug research led to an FDA-approved product with strong commercial uptake, but its other neuroscience efforts have yet to achieve the same outcomes. […]
A Rocket Pharmaceuticals gene therapy for the rare immune disorder leukocyte adhesion deficiency-I has met the survival goals of a pivotal study, paving the way […]
In collaboration with leading integrin drug developers, including Genentech, 7 Hills Pharma, and Pfizer, the 2nd Integrin-Targeted Drug Development Summit is designed for industry leaders to unite and outline clinical […]
PepGen’s technology improves the delivery of a therapy to more tissue types and the clinical-stage biotech plans to use its IPO cash to continue developing […]
UK-based OMass Therapeutics is leaning on a proprietary form of native mass spectrometry to help develop small-molecule drugs for rare diseases.
Bristol Myers Squibb drug Camzyos has FDA approval for treating obstructive hypertrophic cardiomyopathy, a rare and potentially fatal heart disorder. The drug is projected to […]
If you were a drug manufacturer, you naturally would want to focus your research on more common, highly prevalent diseases. If there are more patients […]
A Novartis drug that stops abnormal cell growth in breast cancer now has an additional FDA approval treating a group of rare disorders characterized by […]
Evozyne uses artificial intelligence and machine learning to design novel proteins that can be used in gene therapies. The new agreement with Takeda Pharmaceutical covers […]
Marinus Pharmaceuticals drug Ztalmy is now FDA approved for treating the seizures in patients who have CDKL5 deficiency, an inherited form of epilepsy. The drug […]
Targeting mutations isn’t the only way to treat genetically driven diseases. Scenic Biotech is developing molecules to target genes that suppress disease, and it has […]
An experimental hemophilia A therapy from Sanofi and partner Sobi has met the goals of a pivotal study, and the pharmaceutical giant is now planning […]
To reduce time-consuming manual searches and improve their findings, many organizations are turning to artificial intelligence technologies such as natural language processing. Clinical and research […]
A new study has suggested that the economic burden posed by rare diseases is around 10 times higher than for common “mass market” conditions. The […]
This year on Rare Disease Day, we join families and communities around the world building awareness around rare diseases and encouraging patients to share their […]
With over 40% of all medicines in the pipeline aimed at rare diseases, the future looks positive for increasing the number of treatments available. However, […]
Orphazyme’s hopes of getting EU approval for its Niemann-Pick disease type C (NPC) therapy arimoclomol after a rejection by the US FDA last year are […]
The SPAC merger that Amicus Therapeutics planned for its gene therapy assets has been called off due to market conditions. The rare disease company is […]
Agios Pharmaceuticals has its third FDA-approved product, and the first under its new rare disease strategy. The regulatory decision also marks the first approved treatment […]
SpliceBio will use its €50 million in financing to advance development of a gene therapy for Stargardt disease, a rare eye disorder. The biotech’s technology […]
By JESSICA DaMASSA, WTF HEALTH Komodo Health has been catching lots of buzz lately thanks to a recently announced partnership with the Chan Zuckerberg Initiative’s […]
A rare disease drug that Sanofi added via its $11.6 billion Bioverativ acquisition is now approved for treating the blood disorder cold agglutinin disease. The […]
Real-World Evidence 2022: Rare Diseases and Innovative Therapies (RWE 2022) will convene the full spectrum of stakeholders working in RWE in rare diseases and innovative […]
The FDA rejected the new drug application for a Levo Therapeutics treatment for the rare metabolic disorder Prader-Willi syndrome. According to Levo, the agency said […]
As in previous years, cancer products topped the list of new FDA-approved therapies in 2021. But the list of drug approvals is notable for other […]
Myasthenia gravis patients now have a new FDA-approved biological treatment. Argenx drug Vyvgart provides a treatment alternative for patients who have the rare autoimmune disorder […]
Today, PhRMA released a new report detailing orphan drug development in the U.S. and the potential to meet the significant unmet medical need for patients. […]
While #Covid19 has impacted populations across the globe, it may have had an even greater effect on patients suffering from #rarediseases. The #pandemic has disrupted […]
Nephrology products company Vifor Pharma is shelling out about $252 million combined up front to acquire Sanifit Therapeutics and Inositec, developers of drugs for a […]
Overcome the Challenge of Treating Unmet Medical Needs Caused by Mitochondrial Dysfunction The 2nd annual Mitochondria-Targeted Drug Development Summit returns as the only industry-led meeting focused on overcoming […]
Shots: Ginkgo is eligible to receive up front, R& D fees including milestones in the form of Selecta common stock, clinical & commercial milestones of […]
The FDA refused to review Stealth BioTherapeutics’ Barth syndrome drug, telling the company results in a study of just eight patients are insufficient to support […]
Far more common than initially thought, rare disease treatments remain far too scarce. The answer to the problem, of course, lays in making medical research […]
Mirum Pharmaceuticals drug Livmarli is now approved to treat pruritus caused by Alagille syndrome, a rare liver disease that can worsen to the point of […]
Amicus Therapeutics is spinning its gene therapy pipeline into a separate entity that will go public via a SPAC merger. Executives say the transaction puts […]
Capricor Therapeutics’ cell therapy for Duchenne muscular dystrophy posted encouraging results from a Phase 2 study enrolling 20 patients. But the FDA said a Phase […]
The third generation of his family to hold a leadership role at Chiesi Group, Giacomo Chiesi reflects on the lessons he learned from his father […]
Global Gene’s Craig Martin introduces a new patient identification initiative that strives to improve diagnosis and access to clinical trials for all patients with rare […]
AI and precision medicine are both changing drug discovery and development as we know it. Precision medicine that is powered by AI stands to be […]
The European Commission has approved a BioMarin Pharmaceutical drug that treats achondroplasia, a rare genetic disorder that slows bone growth and is the most common […]
The FDA has approved Bylvay, an Albireo Pharma drug that treats the severe itching experienced by patients with progressive familial intrahepatic cholestasis (PFIC). Prior to […]
The EU medicines regulator has said that there is some evidence to suggest a possible link between mRNA-based vaccines for COVID-19 and rare cases of […]
Takeda’s Adam Zaeske discusses how cross-sector collaboration is shaping a brighter future for rare disease diagnosis. Diagnosis can sometimes be the most difficult part of […]
Orphazyme’s corporate restructuring comes less than two weeks after the FDA rejected arimoclomal, a drug developed to treat the rare Niemann-Pick disease type C. The […]
Intellia Therapeutics has early clinical trial data showing that its CRISPR-based therapy for a rare disease can edit genes inside the body safely and effectively. […]
Auris Medical has acquired Trasir Therapeutics, a startup developing technology that expands the delivery of RNA medicines to tissues beyond the liver. Going forward, Auris […]
The FDA placed a clinical hold on a Larimar Therapeutics program for Friedreich’s ataxia after monkeys died in tests of the fusion protein. In addition […]
A Biogen gene therapy for a rare, inherited eye disorder that leads to blindness has failed a key clinical trial. The preliminary results are the […]
Real-world data is a vital part of rare disease drug development, but to get a true picture of patients’ unmet needs pharma should take a […]
Protracted diagnostic odysseys don’t only block access to rare disease treatments, they also leave people facing emotional turmoil all alone. Growing up in Glasgow, Linsey […]
Alnylam Pharmaceuticals has filed for FDA approval of vutrisiran, which could become the biotech’s fourth drug that works by RNA interference. Meanwhile the company is […]
On Wednesday, April 28th, the World Orphan Drug Congress USA is being held online for free! The conference brings together pharma, biotechs, government, payers, investors […]
Roche’s Genentech unit has released further data from a key trial of Evrysdi, its oral treatment for spinal muscular atrophy (SMA), the rare muscle wasting […]
Alexion’s senior vice president of its international business talks to Paul Tunnah about the importance of “people-centred and vulnerable” leadership, the surprising impacts of COVID […]
What is a rare disease? The definition varies across countries, but according to the European Medicines Agency (EMA) rare diseases are those with a prevalence […]
Design Therapeutics raised $240 million in the biggest biotech IPO this week. One other rare disease drug developer went public along with two oncology biotechs […]
ProQR Therapeutics has encouraging data from an early-stage study evaluating its treatment for vision loss in the rare, inherited disorder Usher syndrome. The biotech now […]
Approximately 85% of rare diseases are genetic, offering significant opportunities to develop better treatments as our understanding of the human genome improves1. Advances in next-generation […]
Approximately 85% of rare diseases are genetic, offering significant opportunities to develop better treatments as our understanding of the human genome improves1. Advances in next-generation […]
Bluebird bio said independent analyses found that the gene therapy inserted its payload at a site not known to cause cancer. Based on the results, […]
While Covid has made many aspects of living with a rare disease more difficult, it has inspired policies and techniques that could help rare disease […]
Takeda Pharmaceutical gains full rights to a small molecule that has shown promise treating seizures in several rare forms of epilepsy. Meanwhile, Ovid Therapeutics gains […]
Rare disease families need to stand up and be counted if they want things to change said mum and advocate Christine Waggoner. She spoke to […]
The FDA decision makes Nulibry the first drug approved to treat MoCD type A, a rare metabolic disease. It’s also the first FDA-approved product for […]
Rare diseases impact between 3.5%-5.9% of the world’s population. This Rare Disease Day, RBW Consulting’s Emma Thorp discusses why the consultancy is taking revenue earned […]
The FDA decision gives Sarepta Therapeutics its third approved drug for Duchenne muscular dystrophy. The accelerated approval requires the biotech to conduct additional clinical testing […]
How has rare disease care changed over the last few years? And what might we expect from the next five? We attended the Rare Disease […]
To help pharma companies understand what life is really like for patients with a rare disease, a new resource has been released to coincide with […]
In the past year, Evox Therapeutics has inked alliances with two big pharma companies. The Series C financing now positions the biotech to advance to […]
Advocacy groups play a crucial role in both driving access to innovative treatment and services and educating patients and caregivers on novel breakthroughs. On average, […]
Launching a rare disease patient registry often requires patient advocacy groups to design a registry themselves or pay for an expensive customized solution. With the […]
Welcome to Delta – the new Fishawack Health magazine. We’ve chosen to focus our first edition of the magazine on a topic close to our […]
The COVID-19 pandemic has added a new layer of complexity to the drug development process. Launch timelines have been delayed and clinical trials have been […]
A recent analysis by Health Advances looks at two particularly burdensome hematological (or blood) diseases — beta thalassemia and hemophilia A — and finds that potential […]
US biotech bluebird bio has announced plans to split into two this year, with a separate oncology business spinning off as the company prepares to […]
Episode four of Kantar Health’s Health Heroes podcast tackles pharma’s rare disease challenges, the role of patient registries and hears a moving story about the […]
Say Goodbye to the Worry & Uncertainty of Setting Up & Managing an Expanded Access Program This inaugural Operationalize: Expanded Access Programs digital event is here to […]
Shares in Rocket Pharmaceuticals have been living up to their name, shooting up following encouraging early-stage clinical trial results from a gene therapy for a […]
Delayed diagnosis, misdiagnosis, and no cure or disease modifying treatments – children living with mitochondrial disease face all the issues often associated with rare diseases. […]
Cure Rare Disease’s Rich Horgan discusses the importance of preclinical NAbs screening and helping rare disease patients better understand their eligibility for gene therapies. Neutralising […]
Amanda Barrell explores how a perfect storm of changing economics, advances in technology, and the increasing volume of the patient voice is stoking change in […]
The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. Oxlumo […]
The FDA has agreed to expedite development of two rare disease drugs from Sanofi. The most significant of the two announcements is the Priority Review […]
Sanofi’s attempt to take on Roche with a ground-breaking approach to treating haemophilia has hit a setback after the company voluntarily placed its late-stage trials […]
Swedish biotech Calliditas has announced plans to file its rare disease drug Nefecon with the FDA early next year following positive results from a phase […]
The UK’s drug cost-effectiveness body NICE has launched a public consultation, presenting the case for change about how it assesses medicines, medical devices and diagnostics. […]
Ring Chromosome 20 Syndrome, or (R)20, is an ultra-rare form of epilepsy with a devastating impact – yet despite huge leaps forward in gene sequencing […]
In the age of artificial intelligence, no trial data should be going to waste. Findacure’s Rick Thompson looks at how these technologies could bring us […]
Delayed diagnosis, lack of awareness, and a limited evidence base – Aarskog syndrome faces all the challenges of rare diseases everywhere. But a new study […]
The post Shining a light on an ‘embarrassing’ condition appeared first on Healthy Debate.
US biotech BioMarin has hit back with a filing for a rare disease drug, after suffering what was described by analysts as a “major setback” […]
The UK-based AI technology company Healx has begun a partnership with the Foundation for Angelman Syndrome Therapeutics (FAST), focusing on developing new therapies for the […]
The FDA has approved a new therapy for the rare muscle wasting disease Duchenne muscular dystrophy (DMD) as Japan’s NS Pharma takes on Sarepta and […]
New research looks at the factors that speed up and slow down HTA appraisals for rare disease medicines across Europe. Rare diseases drugs have always […]
Biogen is planning to trial its Spinraza spinal muscular atrophy (SMA) drug in patients who have not responded properly to Novartis’ gene therapy Zolgensma, combining […]
The COVID-19 pandemic has bit into sales at Novartis, the big Swiss pharma said in second quarter results, after orders fell back following a period […]
US biotech Inozyme Pharma is the latest to jump on to the IPO bandwagon, setting terms for a $75 million stock market launch, as the […]
SMi reports: Dr. Thakur Raghu Raj Singh from Queen’s University Belfast will lead the post conference workshop on Wednesday 25th November 2020. SMi presents the […]
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