The FDA has granted fast track designation to Solid Biosciences’s gene therapy SGT-003 for the treatment of DMD. The US-based company received clearance from the FDA to start Phase I/II trial for the gene therapy on 14 November. SGT-003 uses a novel capsid AAV-SLB101 to deliver the DNA sequence encoding the shortened form of the dystrophin protein (microdystrophin). It also has R16 and R17 nNOS binding protein domains. DMD is a rare genetic condition that causes progressive muscle weakness. It affects approximately six in 100,000 individuals across Europe and North America, as per the US Muscular Dystrophy Association. The first gene therapy for treating DMD was approved earlier this year. In June, Sarepta Therapeutics’s gene therapy Elevidys (delandistrogene moxeparvovec-rokl) received accelerated approval by the FDA for treating ambulatory patients aged 4-5 years with a confirmed mutation in the DMD gene. There has been an increased interest in developing gene therapies ...
Novartis’ Fabhalta (iptacopan) has been approved by the US Food and Drug Administration (FDA) as the first oral monotherapy for adults with paroxysmal nocturnal haemoglobinuria (PNH), a rare blood disease affecting approximately ten to 20 people per million worldwide. PNH patients have an acquired mutation that causes them to produce red blood cells susceptible to premature destruction by the complement system, which can cause anaemia, thrombosis, fatigue and other symptoms that can impact quality of life. The disease has a significant unmet need, Novartis reports, with a large proportion of patients on anti-C5 treatment remaining anaemic and dependent on blood transfusions. Fabhalta is a factor B inhibitor of the immune system’s complement pathway and offers an alternative to therapies that need to be delivered by infusion or injection. Victor Bultó, president US, Novartis, described the approval as an “extraordinary moment” for the PNH community. “This new, effective oral medicine may ...
The European Commission (EC) has approved UCB’s Zilbrysq (zilucoplan) as an add-on therapy for certain patients with generalised myasthenia gravis (gMG), a rare autoimmune disease with a global prevalence of 100 to 350 cases per one million people. The marketing authorisation, which specifically applies to adults who are anti-acetylcholine receptor (AChR) antibody-positive, makes Zilbrysq the first once-daily subcutaneous, targeted component 5 (C5) complement inhibitor for gMG. Patients with gMG can experience a variety of symptoms, including severe muscular weakness that can result in double vision, drooping eyelids, difficulty swallowing, chewing and talking, and life-threatening weakness of the muscles of respiration. UCB’s Zilbrysq inhibits complement-mediated damage to the neuromuscular junction through its targeted dual mechanism of action, the company said, adding that the therapy can be used simultaneously with intravenous immunoglobulin and plasma exchange without the need for supplemental dosing. The EC’s decision was supported by positive results from the late-stage ...
By Connor Lynch Pictured: A patient being examined by an eye doctor EyePoint Pharmaceuticals marked a major win Monday, with Phase II results of its anti-VEGF therapy for wet age-related macular degeneration showing comparable results to Regeneron’s Eylea on a less-frequent dosing regimen. Share prices jumped over 200% in premarket trading on the news that EYP-1901 had hit all primary and secondary endpoints in the DAVIO 2 trial. The trial was investigating EyePoint’s EYP-1901 therapy, an “investigational sustained delivery maintenance treatment” for wet age-related macular degeneration (AMD) that combines vorolanib, a selective tyrosine kinase inhibitor, with “bioerodible Durasert E,” an injectable drug delivery system. The study tested two doses of the therapy, both 2 mg and 3 mg in 160 patients, finding both met all of the primary and secondary endpoints. That included the primary endpoint of “statistical non-inferiority change in best corrected visual acuity,” compared to the control of ...
US-based Generation Bio is cutting its workforce by 40%, becoming the latest company to announce lay-offs to save money this year. The restructuring includes the departure of key personnel, with medical chief Douglass Kerr and development leader Tracy Zimmerman among those leaving. The 29 November announcement adds to a series of setbacks in the field, with the cell therapy startup NexImmune reducing its workforce by over half this year, halting the development of its three adoptive T cell therapies. Generation anticipates that the downsizing and streamlining of its research and development focus will result in a cost-saving of $120m over the next three years, as outlined in a 27 November SEC filing. In April, Takeda announced that it was moving away from the adeno-associated virus (AAV) gene therapies sector amidst difficulties associated with gene therapy research development at the preclinical stage, highlighting that it is unlikely that many current AAV ...
AstraZeneca’s rare disease unit has received a recommendation from the National Institute for Health and Care Excellence (NICE) for the use of its enzyme replacement therapy in infants with Wolman disease. Alexion’s Kanuma (sebelipase alfa), which has been specifically recommended for use in patients who are aged two years or younger when administration begins, will now become the first treatment available on the NHS for the rapidly-progressive rare genetic disease. Occurring in around one in 350,000 births, Wolman disease causes a build-up of fat in cells in the liver, heart, blood vessels and digestive system. Symptoms in infants include enlarged liver and spleen, poor weight gain, low muscle tone, jaundice, vomiting, diarrhoea, developmental delay and anaemia. Until now, standard care for the disease has been palliative and limited to managing symptoms, with patients normally not surviving past the age of one without treatment. Administered as weekly intravenous infusions which can ...
British biopharmaceuticals company Autolus Therapeutics has submitted a Biologics License Application (BLA) to the Food and Drug Administration (FDA) for its chimeric antigen receptor T cell therapy (CAR-T therapy), which treats a persistent form of leukaemia. The therapy, obe-cel, targets relapsed/refractory adult B-cell acute lymphoblastic leukaemia (ALL), a blood cancer that currently has survival rates ranging from <10% to around 25% across all age groups. These rates are even lower for adults in older life. According to Phase II trial data, Autolus’ treatment had an overall response rate of 76%, with 97% of responders becoming minimal residual disease (MRD)-negative, meaning they entered a complete remission. After a median follow-up of nine and a half months, 61% of responders remained in remission without subsequent anti-cancer therapies. These results have allowed the drug to obtain Orphan Drug Designation from the FDA and Orphan Medical Product Designation from the European Medicines Agency (EMA). ...
I work as a cardiologist for a multi-specialty group that sees patients both in capitated payer and fee-for-service environments. This diverse practice gives me the experience to know where insurance providers will find value or what they will likely pay for. As these protocols become more scientifically validated, AI is going to help the entire healthcare system to identify at-risk patients quickly and accurately. By DR. JONATHAN A. ALIOTA As a cardiologist, I see this kind of scenario play out on a daily basis. A patient comes in for a routine procedure and a heart murmur is detected. This murmur could have several different diagnoses. While they can be a sign of something more serious, many of the heart murmurs we hear on a routine basis are normal, physiologic murmurs, such as mild mitral regurgitation. In addition, a healthy heart that had too much caffeine and not enough water that ...
Health Canada has approved Jazz Pharmaceuticals’s Epidiolex (cannabidiol oral solution) as an adjunct therapy in patients aged two years and older for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome, and tuberous sclerosis complex (TSC). Epidiolex is a plant-based oral cannabidiol solution. It was developed by GW Pharmaceuticals and was added to Jazz’s portfolio following GW’s acquisition in 2021. The therapy was first approved in the US as an adjunct therapy in patients aged two years and older for the treatment of seizures associated with LGS and Dravet syndrome in June 2018. The approval in TSC came in July 2020. All three indications causes different types of serizure. LGS and Dravet syndrome are types of epileptic encephalopathy, that cause seizures. LGS is managed by two or more seizure medications whilst Dravet is treatment-resistant. TCS is a rare genetic condition that causes benign tumours to develop in different ...
The US Food and Drug Administration (FDA) has granted a fast track designation to Alladapt Immunotherapeutics’ IgE-mediated multi-food oral immunotherapy ADP101. The FDA’s decision was based on the results from the Phase I/II Harmony trial, which investigated the immunotherapy’s use in inducing desensitisation in patients with single or multiple food allergies, including nuts, dairy, seafood and more. Fast track designations enable earlier interactions with the FDA for the pursuit of accelerated approval. The status also opens chances of rolling and priority reviews. In this instance the designation allows Alladapt to seek accelerated approval for oral immunotherapy in paediatric patients, aged between four and 17 years, with one or more of the 15 food allergies covered by ADP101, as per a 22 November press release. Phase I/II data The double-blind, placebo-controlled Phase I/II Harmony trial (NCT04856865) enrolled 61 paediatric patients (aged four-17 years) and 12 adult patients (aged 18-55 years). The ...
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