Rare Diseases
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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
Pharmaceutical
Japanese drug major Eisai (TYO: 4523) says that the European Commission has approved for marketing its Fycompa (perampanel) as an adjunctive treatment of partial-onset seizures, with or without secondarily generalized seizures, in people with epilepsy aged 12 years and older. With the EC's approval, the European Union is the first region in the world to approve Eisai's perampanel. 30 July 2012
Biotechnology
US RNA-based drug developer Sarepta Therapeutics (Nasdaq: SRPT) saw its shares leap 140% to $8.30 yesterday after the company, formerly known as AVI BioPharma, announced that treatment with its exon-skipping compound, eteplirsen, achieved a significant clinical benefit on the primary clinical outcome, the 6-minute walk test (6MWT), over a placebo/delayed treatment cohort in a Phase IIb trial in patients with Duchenne muscular dystrophy (DMD), a devastating progressive, degenerative, universally fatal neuromuscular disorder that affects about one in every 3,500 boys worldwide. 25 July 2012
Biotechnology
In the UK, the role of assessing very high cost drugs for people who suffer with rare conditions, under the Health and Social Care Act 2012, currently managed by the Advisory Group for National Specialised Services (AGNSS), will pass to the country's main drugs watchdog, the National Institute for Health and Clinical Excellence (NICE), which has long been criticized by pharmaceutical companies for keeping expensive medicines out of the reach of National Health Service patients. 25 July 2012
Biotechnology
Reversing an earlier negative opinion (The Pharma Letter April 22), the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) on Friday issued a positive opinion that recommends marketing authorization of Netherlands-based uniQure's Glybera (alipogene tiparvovec) as a treatment for lipoprotein lipase deficiency (LPLD). 23 July 2012
Biotechnology
Moving forward with its foray into the rare disease sector, UK pharma giant GlaxoSmithKline (LSE: GSK) and USA-based biotech firm Amicus Therapeutics (Nasdaq: FOLD) expanded their collaboration to develop and commercialize the investigational pharmacological chaperone migalastat HCl, trade name Amigal, for Fabry disease. The expanded alliance comprises three components: 19 July 2012
Biotechnology
US company Auxilium Pharmaceuticals (Nasdaq: AUXL) and the Canadian subsidiary of Actelion (SIX: ATLN), Europe's largest biotech firm, have received marketing approval from Health Canada for Xiaflex (collagenase clostridium histolyticum or CCH), a novel, first-in-class biologic for the treatment of Dupuytren's contracture in adults with a palpable cord. 10 July 2012
Pharmaceutical
Eltrombopag, a drug that was designed to stimulate production of platelets from the bone marrow and thereby improve blood clotting, can raise blood cell levels in some people with severe aplastic anemia who have failed all standard therapies. 5 July 2012
Biotechnology
US biotech firm Chelsea Therapeutics International (Nasdaq: CHTP) shares tumbled in pre-market trading yesterday, after it received a written response from the Food and Drug Administration to the company's modified proposal for utilizing data from ongoing Study 306B to support its application for marketing approval of Northera (droxidopa) in the USA as a treatment for symptomatic orthostatic hypotension (SOH). The stock, already down significantly this year, fell a further 51% to $0.71. 4 July 2012
Biotechnology
In a second accord between the US biotech companies this year, Biogen Idec (Nasdaq: BIBB) and Isis Pharmaceuticals (Nasdaq: ISIS) have entered into an exclusive, worldwide option and collaboration agreement under which they will develop and commercialize a novel antisense drug for the treatment of myotonic dystrophy type 1 (DM1), also known as Steinert disease. 2 July 2012
Pharmaceutical
USA-based Vertex Pharmaceuticals (Nasdaq: VRTX) saw its shares tumble 16% to $51.18 last week, after the company announced final data from a Phase II study of VX-809 and Kalydeco (ivacaftor) that showed statistically significant improvements in lung function among adults with cystic fibrosis (CF) who have two copies (homozygous) of the most common mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, F508del. 1 July 2012
Pharmaceutical
Japanese drugmaker Teijin Pharma (TYO: 3402) revealed last Friday that it has received manufacturing and marketing approval for Somatuline (lanreotide acetate) subcutaneous injection for the treatment of acromegaly and pituitary gigantism from Japan's Ministry of Health, Labor and Welfare. 1 July 2012
Biotechnology
Bayer HealthCare, a US unit of Germany's Bayer (BAYN: DE) and partner Onyx Pharmaceuticals (Nasdaq: ONXX) said yesterday that the US Food and Drug Administration has granted priority review designation for the New Drug Application filed end of April 2012 for the oral multi-kinase inhibitor regorafenib, for the treatment of patients with metastatic colorectal cancer (mCRC) whose disease has progressed after approved standard therapies. 29 June 2012
Pharmaceutical
Shire's Vpriv shows significant improvement in Gaucher-related bone disease 28 June 2012
Pharmaceutical
Following meetings last week, the European Medicines Agency issued a number of recommendations and opinions regarding marketing authorization applications from Nycomed, AstraZeneca, Novartis, Pfizer and Sanofi Pasteur. 25 June 2012
Pharmaceutical
Swiss drug major Roche (ROG: SIX) and privately-held US firm Seaside Therapeutics have entered into a collaboration to develop disease modifying treatments for fragile X syndrome (FXS) and autism spectrum disorders (ASD), both neurodevelopmental disorders for which there are currently no effective pharmacological treatments that address core symptoms. 20 June 2012
Pharmaceutical
The US Food and Drug Administration yesterday issued a complete response letter regarding drugs behemoth Pfizer's (NYSE: PFE) New Drug Application for tafamidis meglumine, requesting the completion of a second efficacy study to establish substantial evidence of effectiveness prior to an approval. The Agency has also asked for additional information on the data within the current tafamidis NDA. 19 June 2012
Biotechnology
French late-stage biopharma company ERYTECH Pharma says that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency has adopted a positive opinion to grant Orphan Drug Designation to its investigational product Enhoxy for sickle cell disease. 14 June 2012
Biotechnology
Netherland-based biotech company Pharming Group (NYSE Euronext: PHARM) yesterday revealed that it is reviewing its strategic options and has engaged Roth Capital Partners and Nomura Code to assist it in exploring alternatives and provides an update on Study 1310 for Ruconest (INN conestat alfa), a recombinant version of the human protein C1 inhibitor (C1INH). 13 June 2012
Pharmaceutical
Israel-based Protalix BioTherapeutics (TASE:PLX) has received a $25 million milestone payment from global pharma behemoth Pfizer (NYSE: PFE), which was triggered by the recent approval of Elelyso (taliglucerase alfa) by the US Food and Drug Administration's approval for the treatment of type 1 Gaucher disease (The Pharma Letter May 2). 13 June 2012
Biotechnology
According to a European Commission statement, over 30 million European citizens suffer from a rare disease. The small numbers of patients affected by a single disease and the fragmentation of knowledge about rare diseases translates into difficulties for people to get the right diagnosis, the right medical advise and the right medicine. 10 June 2012
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