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Pharmaceutical
Quebec-based Medunik Canada, a unit of family-owned Groupe Pharmaceutique Boivin, has concluded a strategic collaboration agreement with QOL Medical of the USA. Under this accord, Medunik receives the exclusive Canadian rights to make available and distribute to patients and physicians two important therapies for individuals suffering from specific rare diseases: Sucraid (sacrosidase) and Ethamolin (ethanolamine oleate). Financial terms of the deal were not disclosed. 29 November 2011
Pharmaceutical
Swiss drug major Novartis' (NOVN: VX) Ilaris (canakinumab), a treatment for cryopyrin-associated period syndrome (CAPS) which gained marketing approval in Japan a couple of months ago (The Pharma Letter September 28), has now received National Health Insurance (NIH) price listing from the Central Social Insurance Medical Council (CSIMC; Chuikyo). 23 November 2011
Pharmaceutical
UK and USA-based specialty drug firm EUSA Pharma says that the US Food and Drug Administration has approved its orphan drug Erwinaze (asparaginase Erwinia chrysanthemi) for the treatment of acute lymphoblastic leukemia (ALL) in patients with hypersensitivity to E. coli-derived asparaginase. 22 November 2011
Pharmaceutical
Following a positive opinion from an advisory committee in the summer (The Pharma Letter July 23), the European Commission has now granted final approval for US drug behemoth Pfizer's (NYSE: PFE) Vyndaqel (tafamidis) for the treatment of the rare and fatal neurodegenerative hereditary disease transthyretin familial amyloid polyneuropathy (TTR-FAP) in adult patients with stage 1 symptomatic polyneuropathy. 18 November 2011
Biotechnology
USA-based Incyte (Nasdaq: INCY) saw its shares rise 10% to$13.90 on the news that the Food and Drug Administration had approved Jakafi (ruxolitinib), the first drug cleared to specifically treat patients with the bone marrow disease myelofibrosis (MF), ahead of the Prescription Drug User Fee Act (PDUFA) target date of December 3. 17 November 2011
Pharmaceutical
USA-based Dyax Corp (Nasdaq: DYAX) says it reached agreement with Defiante Farmaceutica, a unit of privately-held Italian drugmaker Sigma Tau, to withdraw the Marketing Authorization Application (MAA) filed with the European Medicines Agency for Kalbitor (ecallantide) in the treatment of hereditary angioedema (HAE). 15 November 2011
Pharmaceutical
Italian drugmaker Recordati (REC: MI) and wholly-owned subsidiary Orphan Europe announced that Normosang, or human hemin, specifically developed to treat acute attacks of hepatic porphyrias, is now registered and approved for use in Russia. 8 November 2011
Pharmaceutical
The oral drug ivacaftor (VX-770) provides major, sustained improvement in lung function, growth and other signs and symptoms for cystic fibrosis (CF) patients, according to results of the pivotal Phase III clinical trial published in the New England Journal of Medicine. The multi-site study marks the first time a drug has been shown to work on the disease process rather than on the symptoms. CF, which has no cure, is the most common lethal genetic disease in Caucasians. 6 November 2011
Pharmaceutical
UK-based ImmuPharma plc (LSE:IMM), which last month regained rights to its investigational lupus drug candidate Lupuzor (IPP-201101) from Cephalon, now part of Teva (The Pharma Letter October 24) has provided an update on the drug's development status. 3 November 2011
Biotechnology
USA-based Biogen Idec (Nasdaq: BIIB) and Portola Pharmaceutical have entered into an exclusive, worldwide collaboration and license agreement under which both companies will develop and commercialize highly selective, novel oral Syk inhibitors for the treatment of various autoimmune and inflammatory diseases, including rheumatoid arthritis (RA) and systemic lupus erythematosus (SLE). 28 October 2011
Pharmaceutical
ViroPharma to acquire DuoCort Pharma for up to $164 million, adding European orphan drug to pipeline
The USA's ViroPharma (Nasdaq: VPHM) says it has signed a definitive agreement to acquire DuoCort Pharma AB, a private company based in Helsingborg, Sweden, focused on improving glucocorticoid replacement therapy. The acquisition of DuoCort will further expand ViroPharma's orphan disease commercial product pipeline. 28 October 2011
Pharmaceutical
Driven primarily by the launch and uptake of Human Genome Sciences (Nasdaq: HGSI) and GlaxoSmithKline's (LSE: GSK) Benlysta (belimumab), the systemic lupus erythematosus (SLE) drug market will increase more than seven-fold, from around $300 million in 2010 to more than $2.1 billion in 2020 in the seven leading markets of the USA, France, Germany, Italy, Spain, the UK and Japan, according to a new report from advisory firm Decision Resources 26 October 2011
Pharmaceutical
Danish drugmaker Lundbeck (LUND: DC) yesterday received approval from the US Food and Drug Administration for Onfi (clobazam) as adjunctive therapy for seizures associated with Lennox-Gastaut syndrome (LGS) in patients two years and older. Onfi will be available in US pharmacies in early January 2012 and is a federally controlled schedule four substance (C-IV), the company said. 25 October 2011
Biotechnology
As part of the ongoing review and re-prioritization of its R&D pipeline, biotech firm Merck Serono, a unit of Germany's Merck KGaA (MRK: DE), has decided to return all rights for safinamide to Switzerland-based Newron Pharmaceuticals (SIX: NWRN). 24 October 2011
Pharmaceutical
USA-based Vertex Pharmaceuticals (Nasdaq: VRTX) says it has filed a New Drug Application with the Food and Drug Administration for Kalydeco (VX-770, ivacaftor), a medicine in development that targets the defective protein that causes cystic fibrosis (CF). 20 October 2011
Pharmaceutical
Global drugs behemoth Pfizer (NYSE: PFE) has teamed up with Iceland-based genomics firm deCODE genetics, with the objective to discover sequence variants associated with specific clinical phenotypes related to systemic lupus erythematosis by utilizing deCODE's expertise in gene discovery. Financial terms of the collaboration have not been disclosed. 14 October 2011
Biotechnology
Global drugs behemoth Pfizer (NYSE: PFE) has made another foray into the rare disease therapy sector, negotiating an exclusive worldwide rights to US privately-held biotech firm GlycoMimetics' investigational compound GMI-1070. The compound is a pan-selectin antagonist currently in Phase II development for the treatment of vaso-occlusive crisis associated with sickle cell disease. 12 October 2011
Biotechnology
The US National Organization for Rare Disorders (NORD) yesterday released a landmark report documenting flexibility in the Food and Drug Administration review of potential treatments for patients with rare diseases. 11 October 2011
Pharmaceutical
Despite an increase in the number of approved orphan drugs in Europe, the overall growth in cost of these medicines as a proportion of total pharmaceutical expenditure is likely to be sustainable over the next decade, according to the results of a new analysis published in the Orphanet Journal of Rare Diseases. 6 October 2011
Pharmaceutical
UK Health Secretary Andrew Lansley this week announced help for patients with rare diseases, whose doctors may not be able to provide them with unlicensed or 'off label' medication. Most drugs are licensed for use in conditions where the drug manufacturers are able to recruit enough patients into clinical trials and where they will yield enough sales to justify running the trial, he said. This presents a consistent problem for patients with rare diseases, whose access to medicines is limited because too few patients are available to run a clinical trial. 6 October 2011
Pharmaceutical
Japanese mid-size drugmaker Otsuka Pharmaceutical announced that it has entered into an exclusive worldwide licensing agreement with Ionis Pharmaceuticals for rights to manufacture and market Ionis' ulefnersen (ION363), a drug candidate under development for the treatment of patients with amyotrophic lateral sclerosis (ALS) caused by mutation of the fused in sarcoma (FUS) gene. 23 November 2024
Biotechnology
Japanese drugmaker Chugai, which is majority owned by Roche, On Friday revealed that Zenyaku Kogyo has obtained regulatory approval from the Ministry of Health, Labor and Welfare (MHLW), for an additional dosage and administration of an anti-CD20 monoclonal antibody Rituxan (rituximab) intravenous injection 100mg and 500mg, which is co-marketed by both companies, for chronic idiopathic thrombocytopenic purpura (ITP) in children. 23 November 2024
Pharmaceutical
Swiss pharma giant Novartis today announced an upgrade to its mid-term guidance, in advance of its Meet Novartis Management event for investors and analysts in London. 21 November 2024
Biotechnology
US biotech major Amgen has announced that Dr Howard Chang will join the company as senior vice president of research, effective December 16, 2024. 21 November 2024
Biotechnology
Australia’s CSL Limited plans to shut down its Californian R&D facility focused on cell and gene therapies by January 2025, signaling a shift away from ex vivo lentiviral-based technology. 19 November 2024
Biotechnology
Cellular immunotherapy specialist Fate Therapeutics soared as much as 73%, and closed up 12.45 at $2.27 yesterday, after it released early-stage data on its FT819 in lupus. 19 November 2024
Pharmaceutical
US antiviral giant Gilead Sciences on Friday unveiled two-and-a-half-year interim analysis from the ongoing Phase III ASSURE study of its Livdelzi (seladelpar). 16 November 2024
Pharmaceutical
French drugmaker Ipsen announced late-breaking data for Iqirvo (elafibranor 80mg tablets) from an interim analysis of the ongoing open-label extension of the Phase III ELATIVE study at the American Association for the Study of Liver Disease (AASLD) congress. 16 November 2024
Biotechnology
Oxford, UK-based rare respiratory diseases focused AlveoGene today revealed it has been granted a Rare Pediatric Disease designation (RPDD) by the US Food and Drug Administration (FDA) for AVG-002, its novel, inhaled gene therapy for lethal neonatal surfactant protein B (SP-B) deficiency. 15 November 2024
Biotechnology
Shares of US genetic meds company MeiraGTx Holdings were up 7% at $7.12 in early trading after it announced important developments along with its third-quarter 2024 financials, reporting a $0.54 per share loss. 13 November 2024
Biotechnology
Dutch firm Argenx and China’s Zai Lab have received an approval from the National Medical Products Administration (NMPA) for Vyvgart Hytrulo (efgartigimod alfa). 13 November 2024
Pharmaceutical
AstraZeneca and Merck & Co announced that their jointly developed drug Koselugo (selumetinib) has shown positive outcomes in the KOMET Phase III trial. 12 November 2024
Biotechnology
Geron Corporation, a biopharma company focused on telomerase inhibition therapies, has announced a $375 million financing agreement aimed at supporting the commercial rollout of Rytelo (imetelstat). 11 November 2024
Pharmaceutical
The US Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program, according to Sarah Wicks and James Valentine at US law firm Hyman, Phelps & McNamara. 11 November 2024
Biotechnology
Sarepta Therapeutics has announced it will cease development of SRP-5051, an experimental drug for Duchenne muscular dystrophy, citing safety issues, feedback from the Food and Drug Administration, and the shifting Duchenne treatment landscape. 11 November 2024
Pharmaceutical
Research results featured heavily in the news last week, first with Viking Therapeutics releasing promising early-stage data for its investigational medicine VK2738 at the ObesityWeek meeting. US biotech Beam Therapeutics released early-stage data on its sickle cell disease candidate BEAM-101. Also of note, Arcus Biosciences and Gilead Sciences announced promising Phase III results for their TIGIT inhibitor domvanalimab in combination with zimberelimab. Denmark’s Novo Nordisk released financial results that attracted interest. 10 November 2024
Pharmaceutical
Italian pharma major Recordati saw its shares dip 3.75% to 50.10 euros this morning, despite posting a set of strong financial results. 8 November 2024
Biotechnology
Asklepios BioPharmaceutical (AskBio) today announced that AB-1003 (also known as LION-101) has received rare pediatric disease designation and orphan-drug designation from the US Food and Drug Administration (FDA) for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). 7 November 2024
Biotechnology
The global market for Duchenne muscular dystrophy (DMD) therapeutics is projected to grow by $4.4 billion between 2024 and 2028, fueled by artificial intelligence and strong demand for new treatments. 7 November 2024
Pharmaceutical
US drugmaker Sarepta Therapeutic saw its shares gain 2.4% to $127.97 as it reported financial results for the third quarter 2024. 7 November 2024
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