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
Applied Therapeutics saw its stock price climb by over two-thirds after an encouraging update on the US Food and Drug Administration review of govorestat. 19 September 2024
TG Therapeutics yesterday shared new five-year data from the ULTIMATE I & II Phase III trials evaluating Briumvi (ublituximab-xiiy) in patients with relapsing forms of multiple sclerosis (RMS). 19 September 2024
Canada’s NanoVation Therapeutics, a privately-held platform company developing innovative technologies to overcome the barriers of nucleic acid delivery, has announced a multi-year partnership with Danish drugmaker Novo Nordisk. 18 September 2024
AstraZeneca’s Fasenra (benralizumab) has been approved in the USA for the treatment of adult patients with eosinophilic granulomatosis with polyangiitis (EGPA, the UK pharma major announced today. 18 September 2024
US biotech Capricor Therapeutics has entered into a binding term sheet with Japanese drugmaker Nippon Shinyaku for the commercialization and distribution in Europe of Capricor’s lead asset, deramiocel. 17 September 2024
Dystonia is a neurological movement disorder characterized by involuntary muscle contractions that cause repetitive movements, abnormal postures, or twisting. 17 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
The Gastrointestinal Drugs Advisory Committee (GIDAC) of the US Food and Drug Administration (FDA) met on Friday to discuss Intercept Pharmaceuticals’ supplemental New Drug Application (sNDA) for Ocaliva (obeticholic acid, OCA). 16 September 2024
Boehringer Ingelheim today announced that the FIBRONEER-IPF trial met its primary endpoint, which was the absolute change from baseline in Forced Vital Capacity [mL] (FVC) at week 52 versus placebo. FVC is a measure of lung function. 16 September 2024
Oligonucleotide therapeutics are rapidly emerging as a promising class of drugs capable of addressing a variety of genetic and complex diseases by targeting RNA sequences.1 16 September 2024
Thursday was a wretched day for Fulcrum Therapeutics, a US biopharma company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases. 13 September 2024
California, USA-based biotech BridgeBio Pharma saw its shares dip 4% to $29.60 yesterday on disappointing clinical results and the company’s decision to drop an early-stage candidate. 11 September 2024
Santhera Pharmaceuticals has signed an exclusive distribution agreement with GENESIS Pharma for Agamree (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD) in 20 markets in Central and Eastern Europe. 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
Last week’s research news included French pharma major Sanofi releasing mixed Phase III results for it tolebrutinib in progressive multiple sclerosis. Also of note, US vaccine developer Vaxcyte announced strong early-stage trial results for its VAX-31 in pneumococcal disease. On the regulatory front, US drugmaker Travere Therapeutics gained full approval from the US Food and Drug Administration (FDA) for its kidney disease drug Filspari. US cancer drug developer IN8bio announced a prioritization of its R&D pipeline to focus on INB-100 in acute myeloid leukemia, along with a workforce cutback. 8 September 2024
The Institute for Clinical and Economic Review (ICER) has posted its revised Evidence Report assessing the comparative clinical effectiveness of three medicines for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). 6 September 2024
The COVID-19 pandemic continues to hit sales at French drugmaker Ipsen, but the company has shown signs of recovery in its third-quarter figures, sending shares 4% higher on Thursday morning. 22 October 2020
Biogen saw its shares fall 2.36% to $260.82 by mid-morning after it presented financial results for the third quarter of 2020, showing that earnings missed analysts’ expectations. 21 October 2020
Evrysdi (risdiplam) has been approved in Brazil by the National Health Surveillance Agency (ANVISA) for the treatment of spinal muscular atrophy (SMA), the drug’s developer, PTC Therapeutics, said today. 21 October 2020
As the healthcare industry continues to face financial strains brought on by the COVID-19 pandemic, the USA’s Medicaid programs need ways to reduce drug spend. 21 October 2020
Privately-held US biopharma company Synspira Therapeutics today announced that the Cystic Fibrosis Foundation has made equity and structured investments in the company of up to $14 million in additional funding. 20 October 2020
US cancer metabolism specialist Agios Pharmaceuticals late Friday announced the withdrawal of its European Marketing Authorization Application (MAA) for Tibsovo (ivosidenib tablets) for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase-1 (IDH1) mutation. 19 October 2020
USA-based BioSpecifics Technologies announced today that it has entered into a definitive merger agreement under which it will be acquired by Ireland-headquartered Endo International. 19 October 2020
Anglo-Irish rare diseases specialist Amryt Pharma today announced the signing of a distribution agreement with Swixx BioPharma, appointing the Baar, Switzerland-based firm as exclusive distributor of Lojuxta (lomitapide) across 17 jurisdictions in Central and Eastern Europe (CEE). 19 October 2020
At its October meeting, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended 10 medicines for approval. These now go the European Commission, which usually provides a final decision within two to three months. 16 October 2020
Chugai Pharmaceutical says it has filed a new drug application with the Ministry of Health, Labor and Welfare (MHLW) for risdiplam, an oral survival motor neuron-2 (SMN2) splicing modifier, for the treatment of spinal muscular atrophy (SMA). 16 October 2020
US biopharma Vertex Pharmaceuticals has announced that the Phase II study of VX-814 in alpha-1 antitrypsin deficiency has been discontinued based upon safety and pharmacokinetic data. 15 October 2020
The Phase III study CHRONOS-3 evaluating Aliqopa (copanlisib) in combination with rituximab in indolent non-Hodgkin's lymphoma (iNHL) patients (n=458) who have relapsed after one or more prior lines of rituximab-containing therapy has met its primary endpoint of prolonged progression-free survival (PFS). 15 October 2020
Swiss biotech Idorsia has effected a capital increase by way of an at-market rights offering with envisaged gross proceeds of approximately 575 million Swiss francs ($632 million). 12 October 2020
The US Food and Drug Administration (FDA) has granted a rare pediatric disease designation to Italfarmaco Group’s givinostat for the treatment of Duchenne muscular dystrophy (DMD), which allows it an expedited review process. 9 October 2020
Despite the coronavirus pandemic causing chaos on a global scale throughout much of 2020, the US Food a d Drug Administration has strived to limit the impact on much-needed, novel drug approvals. 9 October 2020
UK-based Enterprise Therapeutics revealed today that its novel TMEM16A potentiator portfolio has been fully acquired by Roche and will be developed by the Swiss pharma giant’s Genentech subsidiary. 7 October 2020
After 13 years of research, India's first personalized medicine for Duchenne muscular dystrophy (DMD) has received the green signal from the Drug Controller General of India (DCGI), paving the way for clinical trials. 5 October 2020
Rett syndrome (RTT) is a rare neurodevelopmental disorder, affecting about 1 in 10,000 girls born each year.1 Present at conception, RTT leads to major developmental regression around 6-30 months after birth, with patients typically becoming withdrawn and losing acquired social and motor skills. 5 October 2020