Today, the US Food and Drug Administration approved Miplyffa (arimoclomol), an oral medication for the treatment of Neimann-Pick disease, type C (NPC). 20 September 2024
US neuroscience company BioXcel Therapeutics has announced a new clinical prioritization and an update on its late-stage development programs for the treatment of agitation with BXCL501. 20 September 2024
Stalicla, a privately-held Swiss biotech, today announced that Dr Paulo Fontoura will be joining its drug development team as chief scientific and clinical development advisor. 19 September 2024
US clinical stage CNS drug developer Athira Pharma is taking decisive action following the company’s announcement of disappointing data from the Phase II/III LIFT-AD clinical trial of fosgonimeton to treat Alzheimer’s disease (AD). 18 September 2024
The US Food and Drug Administration (FDA) has approved Ocrevus Zunovo (ocrelizumab and hyaluronidase-ocsq) for the treatment of relapsing multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS), from Swiss pharma giant Roche. 16 September 2024
US pharma major Eli Lilly has announced a $1 billion expansion of its manufacturing site in Limerick, Ireland, to increase production of biologic active ingredients, including those for its recently approved treatment for early symptomatic Alzheimer's disease. 13 September 2024
US neurological diseases specialist Ovid Therapeutics has announced the promotion of Meg Alexander to president and chief operating officer (COO). 12 September 2024
The global migraine market in mature economies is projected to grow from $9.2 billion in 2023 to $16.4 billion by 2033, according to a report from GlobalData. 10 September 2024
Cartesian Therapeutics today announced that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to Descartes-08 for the treatment of juvenile dermatomyositis (JDM). 9 September 2024
California’s Lykos Therapeutics is still trying to regroup after being rocked by the US Food and Drug Administration’s (FDA) rejection of midomafetamine (MDMA) capsules for the treatment of post-traumatic stress disorder (PTSD). 6 September 2024
The cost of prescribed dependency-forming medicines has decreased by £410 million ($539.6 million) since 2015/16, according to newly released data from the UK’s National Health Service (NHS). 6 September 2024
Shares in US biopharma Axsome Therapeutics, a biopharmaceutical company developing and delivering novel therapies for the management of CNS disorders, closed 7% higher on Wednesday. 5 September 2024
Mid-Atlantic BioTherapeutics (MABT) has announced a collaboration with Accelero Biostructures to identify novel inhibitors of USP30, a protein linked to neurological disorders and other conditions. 5 September 2024
Praxis Precision Medicines shared positive top-line results for its Phase II, proof of concept study evaluating relutrigine in SCN2A and SCN8A developmental and epileptic encephalopathy (DEE) patients. 4 September 2024
The US Food and Drug Administration has accepted for review the New Drug Application (NDA) for Azedra (iobenguane I 131) in patients with malignant, recurrent and/or unresectable pheochromocytoma and paraganglioma, which are rare neuroendocrine tumors. 2 January 2018
The US Food and Drug Administration has accepted for filing with Priority Review the recently submitted New Drug Application (NDA) for Epidiolex (cannabidiol or CBD), an investigational treatment for seizures associated with Lennox-Gastaut syndrome (LGS) and Dravet syndrome, two rare and difficult to treat conditions of childhood-onset epilepsy. 28 December 2017
The US Institute for Clinical and Economic Review (ICER) has released a Final Evidence Report and Report-at-a-Glance on vesicular monoamine transporter 2 (VMAT2) inhibitors for management of tardive dyskinesia (TD), saying that the evidence suggests these drugs offer short-term benefits but are ‘vastly overpriced’. 27 December 2017
US biotech major Amgen has published the results from its Phase III trial STRIVE, which evaluated the efficacy of the drug erenumab - in the treatment of episodic migraine and delivered significant differences versus the placebo in all endpoints. 22 December 2017
Japanese and US pharma majors Eisai and Biogen have reported that Study 201, investigating Alzheimer’s candidate BAN2401, has not reached its primary endpoint after 12 months. 21 December 2017
Biogen is to pay $25 million upfront to Ionis Pharmaceuticals as part of an expansion of their collaboration to identify new antisense oligonucleotide drug candidates for the treatment of spinal muscular atrophy (SMA). 20 December 2017
The UK’s National Institute for Health and Care Excellence has today published draft guidance recommending multiple sclerosis drug Extavia (beta 1b interferon), from Swiss pharma giant Novartis, for routine funding on the National Health Service (NHS). 20 December 2017
Two months after filing with the US FDA, embattled generics giant Teva Pharmaceutical Industries’ has been given priority review and fast track designation for fremanezumab, a novel biologic for the preventive treatment of migraine. 19 December 2017
Pharmaceutical companies in India have been providing scientific insights into the genetic causes and biological processes underlying neurodegenerative diseases, reports The Pharma Letter’s India correspondent. 18 December 2017
The USA’s Allergan and Budapest-based Gedeon Richter have announced positive topline results from the Phase III RGH-MD-54 study of once daily atypical antipsychotic Vraylar (cariprazine). 18 December 2017
Carlsbad, California-based Ionis Pharmaceuticals has announced that Roche has licensed its IONIS-HTTRx after positive Phase I/IIa data in Huntington's disease (HD). 13 December 2017
UK-based biopharma firm GW Pharmaceuticals today announced the termination of its previous license agreement with Japan’s Otsuka Pharmaceutical in relation to Sativex (nabiximols) in the USA. 13 December 2017
Drugs that prove to be an effective treatment for migraines could find their way into the hands of as many as 13% of Americans, so it is no surprise that several of pharma’s biggest players are in the race to gain a share of the market. 12 December 2017
It is the third-most common disorder in the world. In the USA, close to 38 million people suffer from it. Globally, the number is a whopping 1 billion. We are talking about migraine, also often referred to as ‘super headaches’ — a condition more prevalent than diabetes, epilepsy, and asthma combined. 11 December 2017
Among last week’s most interesting news were two acquisition deals in the field of immuno-oncology: Gilead Sciences buy of Cell Design Labs and bluebird bio’s takeover of Scottish firm TC BioPharm. Gilead also added to the news flow, in that the first patient has been dosed with its newly-approved lymphoma drug Yescarta by the Moffitt Cancer Center. On the licensing front, Collegium Pharmaceuticals acquired rights to Nucynta and Juno Therapeutics lined up with Eli Lilly, taking the US pharma major into the immuno-oncology arena. 10 December 2017
Shares of CNS drug developer Sage Therapeutics surged yesterday, after the company revealed a second successful clinical trial in as many months, this time with its depressive disorder drug candidate SAGE-217. 8 December 2017
Seven months post-launch, the clear majority of US neurologists (n=103) have initiated at least one patient on Roche (ROG: SIX) subsidiary Genentech's Ocrevus (ocrelizumab), with the brand share being boosted by uptake in both relapsing remitting multiple sclerosis (RRMS) and primary progressive multiple sclerosis (PPMS) patients, new report shows. 8 December 2017
The Australian government announced today that it will invest A$640 million ($485 million) to support Australia’s world-leading health and medical researchers as they continue their work in the laboratory, clinic and the community to find the next major medical breakthrough. 6 December 2017
German family-owned drugmaker Grünenthal has decided to end its partnership with US firm Depomed for the marketing of its opioid pain management product Nucynta (tapentadol). 5 December 2017