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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
Generally unimpressive treatment line-ups combined with weak product pipelines will lead to modest growth for several inherited orphan blood disease markets, states business intelligence provider GBI Research. 23 January 2013
Biotechnology
Netherlands-based biotech firm Dezima Pharma says it has in-licensed a cholesteryl ester transfer protein (CETP) inhibitor DEZ-001 (formerly TA-8995) from Japanese drug major Mitsubishi Tanabe Pharma (TYO: 4508). Financial terms of the deal were not disclosed. 22 January 2013
Pharmaceutical
Teijin Pharma, the core company of the Japanese Teijin Group (TYO: 3401) health care business, and French drugmaker Ipsen (Euronext: IPN), said yesterday that they have launched Somatuline (lanreotide) 60/90/120 mg for subcutaneous injection in Japan for the treatment of acromegaly and pituitary gigantism (when response to surgical therapies is not satisfactory or surgical therapies are difficult to perform). In Japan, Teijin Pharma holds the rights to develop and market the drug. 18 January 2013
Pharmaceutical
R&D, health system capacity-strengthening efforts, and medicine donations comprise the pharmaceutical industry's holistic approach to addressing neglected diseases, according to a new report released yesterday by the International Federation of Pharmaceutical Manufacturers & Associations (IFPMA). The 2012 status report covers pharmaceutical R&D to address neglected diseases that disproportionately affect people in low- and middle-income countries. 16 January 2013
Pharmaceutical
A new study has found that tamoxifen, a well-known, off patent, breast cancer drug, can counteract some pathologic features in a mouse model of Duchenne muscular dystrophy (DMD). At present, no treatment is known to produce long-term improvement of the symptoms in boys with DMD, a debilitating muscular disorder that is characterized by progressive muscle wasting, respiratory and cardiac impairments, paralysis, and premature death. This study will be published in the February 2013 issue of The American Journal of Pathology. 15 January 2013
Pharmaceutical
During its presentation at the JP Morgan annual health care conference, US drugmaker Vertex Pharmaceuticals (Nasdaq: VRTX) revealed that the US Food and Drug Administration had granted the first two Breakthrough Therapy Designations to it Kalydeco (ivacaftor) monotherapy and the combination regimen of VX-809 with ivacaftor for cystic fibrosis. 10 January 2013
Biotechnology
Ireland-headquartered drugmaker Shire (LSE: SHP) said yesterday that it has signed an agreement to acquire privately-held US biotech firm Lotus Tissue Repair, which is developing the first and only protein replacement therapy currently being investigated for the treatment of dystrophic epidermolysis bullosa (DEB). 9 January 2013
Biotechnology
Netherlands and Belgium-based preclinical stage biotech firm arGEN-X, specialized in the discovery and development of highly differentiated human monoclonal antibody products, is broadening its therapeutic antibody collaboration with Ireland-headquartered Shire (LSE: SHP). 8 January 2013
Biotechnology
USA-based Repligen (Nasdaq: RGEN) saw its share jump 5.6% to $6.63, after the company revealed it has signed an exclusive worldwide licensing agreement with global drugs behemoth Pfizer (NYSE: PFE) to advance its spinal muscular atrophy (SMA) program, originally in-licensed from Families of SMA (FSMA). 4 January 2013
Biotechnology
The US Food and Drug Administration has recently approved emerging US biotech firm Aegerion Pharmaceuticals' (Nasdaq: AEGR) Juxtapid (lomitapide) to reduce low-density lipoprotein (LDL) cholesterol, total cholesterol, apolipoprotein B, and non-high-density lipoprotein (non-HDL) cholesterol in patients with homozygous familial hypercholesterolemia (HoFH). 28 December 2012
Pharmaceutical
US drugmaker Vertex Pharmaceuticals (Nasdaq: VRTX) says that the label on its new hepatitis C drug Incivek (telaprevir) in the USA has been updated to include a Boxed Warning stating that fatal and non-fatal serious skin reactions have been reported in patients taking Incivek combination treatment. 20 December 2012
Biotechnology
US biotechnology company ImmunoGen (Nasdaq), which develops anticancer therapeutics using its Targeted Antibody Payload (TAP) technology and antibody expertise, has licensed the exclusive right to use its maytansinoid TAP technology to global independent biotech Amgen (Nasdaq AMGN) to develop anticancer therapeutics to a third target, which is undisclosed. Amgen licensed rights for two other targets in 2009 and has two compounds in clinical testing under those licenses. 20 December 2012
Pharmaceutical
Two US subsidiaries of French drug major Sanofi (Euronext: SAN) have agreed to pay $109 million to resolve allegations that Sanofi US violated the False Claims Act by giving physicians free units of Hyalgan (hyaluronate), a knee injection, in violation of the Anti-Kickback Statute, to induce them to purchase and prescribe the product. 20 December 2012
Biotechnology
The European Medicines Agency's policy on fee reductions for orphan medicinal products is set to continue into 2013. The policy, as advised by the Agency's Committee for Orphan Medicinal Products (COMP), focuses primarily on assistance to micro, small and medium-sized enterprises (SMEs) with the objective of providing incentives to support research and innovation for medicines intended for the diagnosis, prevention or treatment of rare diseases. 19 December 2012
Pharmaceutical
In a flurry of activity from the US Food and Drug Administration on Friday, the agency - which looks likely to set a record of new drug approvals in the current year, issues positive rulings for three more, one of which was three months ahead of expectations. 17 December 2012
Biotechnology
French drug major Sanofi's (Euronext: SAN) subsidiary Genzyme and US partner Isis Pharmaceuticals (Nasdaq: ISIS) have said they plan to request a re-examination of the negative opinion issued last Friday by the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) regarding their drug Kynamro (mipomersen) for the treatment of patients with homozygous familial hypercholesterolemia (HoFH). USA-based Vanda Pharmaceuticals (Nasdaq: VNDA) also received notification that the CHMP does not recommend the firm's Fanaptum (oral iloperidone tablets), which it too plans to appeal. 17 December 2012
Pharmaceutical
Italian drugmaker Recordati *RC: MI) has signed an agreement for the acquisition of all rights concerning a portfolio of products indicated for the treatment of rare and other diseases and marketed mainly in the USA, from Denmark' Lundbeck (LUND: DC). 17 December 2012
Biotechnology
In what has been a busy newsflow few days for USA-based Isis Pharmaceutical (Nasdaq: ISIS), the company said yesterday that the US Food and Drug administration has granted ISIS-TTRRx fast track designation for the treatment of familial amyloid polyneuropathy (FAP). 14 December 2012
Pharmaceutical
Israeli drug developer Protalix BioTherapeutics (NYSE-AMEX: OLX) said yesterday that it has entered into a clinical development agreement with USA-based Pfizer (NYSE: PFE), the world biggest drugmaker by sales, under which Protalix will continue to manage, administer and sponsor current, ongoing clinical trials relating to Elelyso (taliglucerase alfa). 7 December 2012
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