Rare Diseases
Refine Search
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
Noting that only about 200 of the nearly 7,000 diseases classified as rare currently have treatments, the National Organization for Rare Disorders (NORD) has called for a US Food and Drug Administration 'statement of policy' on regulation of therapies for rare disorders. 1 July 2010
Generics
of its June 21-24 meetings, which included several positive opinions to recommend backing approval of new drugs, as well extensions of indications and generic copies. 28 June 2010
Pharmaceutical
Florida, USA-based Biovest International yesterday announced at the Active Immunotherapeutics Forum in Barcelona, Spain, that it is targeting mantle cell lymphoma, in addition to follicular lymphoma, as a next indication for its personalized cancer vaccine, BiovaxID. 25 June 2010
Pharmaceutical
USA-based Dyax Corp has entered a strategic partnership with Defiante Farmaceutica, as subsidiary of Italian drugmaker Sigma-Tau, to develop and commercialize subcutaneous DX-88 (ecallantide) for the treatment of hereditary angioedema (HAE) and other therapeutic indications throughout Europe, North Africa, Middle East and Russia. 22 June 2010
Pharmaceutical
Further confirming its interest in the area of unmet medical needs, global drugs behemoth Pfizer has announced the creation of a new research unit focused on rare diseases. This new unit will significantly expand Pfizer's presence in rare disease research with the goal of discovering novel, life-saving medicines for diseases affecting less than 200,000 patients, the company said. 17 June 2010
Pharmaceutical
Just a day after GlaxoSmithKline received a refusal to fund National Health Service use of its breast cancer drug Tyverb (lapatinib; The Pharma Letter June 10), the UK's National Institute for Health and Clinical Excellence (NICE), in a preliminary guidance, has said it cannot recommend another of its drugs, Revolade (eltrombopag), for treating chronic immune (idiopathic) thrombocytopenic purpura (ITP), a rare blood clotting disorder in splenectomized adults who do not respond to other treatments (for example, corticosteroids, immunoglobulins or as second-line treatment for non-splenectomized adults when surgery is not advised. 11 June 2010
Biotechnology
Troubled US biotechnology firm Genzyme has at last received some good news, with the Food and Drug Administration granting marketing approval for Lumizyme (alglucosidase alfa), the first treatment approved in the USA specifically to treat patients with late-onset Pompe disease, in a decision that came ahead of schedule. 26 May 2010
Biotechnology
Troubled US biotechnology firm Genzyme has signed a consent decree agreeing to correct manufacturing quality violations at its Allston, Massachusetts, manufacturing facility and will turn over to the federal government $175 million in 'unlawful profits' from the sale of products that were made at the plant, the Food and Drug Administration announced yesterday. 25 May 2010
Pharmaceutical
The MDS UK Patient Support Group and the health care professional body, the MDS UK Forum, today presented a 655-signature petition to the UK's new Prime Minister, David Cameron, asking for the same standard of care for UK patients with bone marrow diseases as that available throughout most of Europe. 17 May 2010
Biotechnology
A signing ceremony was held early this week for a strategic partnership agreement between US biotechnology firm Genzyme and China's Tianjin International Joint Academy of Biotechnology and Medicine (TJAB) in the Tianjin Economic-Technological Development Area (TEDA). 14 May 2010
Pharmaceutical
South Africa's Technology Innovation Agency (TIA) yesterday became the first government agency to join in the Pool for Open Innovation against Neglected Tropical Diseases, which was established in February 2009. The TIA intends to use intellectual property and know-how from the pool to accelerate its efforts to grow the South African biotechnology sector and enhance the quality of life of those affected by neglected tropical diseases. Initially, the TIA will focus on developing new medicines for tuberculosis and malaria. 6 May 2010
Biotechnology
In a study published on-line in Blood, the journal of the American Society of Hematology, researchers present positive results of a Phase II clinical trial of eliglustat tartrate, an oral therapy in development to treat Gaucher's disease by US biotechnology firm Genzyme. 5 May 2010
Biotechnology
Troubled US biotech firm Genzyme has advised the European Union health regulator that the supply shortages of its rare disease drugs Cerezyme (imiglucerase) and Fabrazyme (agalsidase beta) are expected to continue at least until the end of September 2010, because of a new manufacturing problem. 23 April 2010
Pharmaceutical
California, USA-based BioMarin Pharmaceutical says that Firdapse (3,4-diaminopyridine) is now commercially available in the European Union for the treatment of the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS). 20 April 2010
Biotechnology
The economic recovery process has proved difficult for the pharmaceutical industry. Factors like patent expiry, dry pipeline and strict approval guidelines, have slowed down the attractive drug discovery and industry growth. Coupled with an investment risk in new therapy or molecules, this creates a challenge for the industry to overcome. However, in the midst of the current status, there seems to be a bend in the road - marked by the evolution of a paradigm shift in the industry: Orphan drugs. 8 April 2010
Pharmaceutical
UK drugs giant GlaxoSmithKline has entered into a new strategic alliance with the USA's Isis Pharmaceuticals for the latter's antisense drug discovery platform to seek out and develop new therapeutics against targets for rare and serious disease, including infectious diseases and some conditions causing blindness. 1 April 2010
Pharmaceutical
USA-headquartered Ikaria Holdings says that it has acquired the New Drug Application (NDA) and the Investigational New Drug (IND) application to Lucassin (terlipressin for injection) from Orphan Therapeutics, assuming all future development and ownership of the drug in North America and Australia. Lucassin being developed for the treatment of hepatorenal syndrome (HRS) Type 1, an orphan-designated condition for which there currently are no approved drugs in the USA. 1 April 2010
Pharmaceutical
The US Food and Drug Administration granted marketing approval for Salix Pharmaceuticals' antibiotic Xifaxan (rifaximin) 550mg tablets for reduction in risk of overt hepatic encephalopathy (HE) recurrence in patients 18 years of age or older. 30 March 2010
Pharmaceutical
California, USA-based Cortex Pharmaceuticals says it has entered into an agreement with Canada's Biovail Laboratories for certain Ampakine compounds and the rights to the patent filing for respiratory depression and vaso-occlusive crises associated with sickle cell disease. The transaction with Biovail includes a purchase price of $10 million and additional payments of up to $15 million on defined clinical development milestones, including approval by the US Food and Drug Administration. 29 March 2010
Biotechnology
UK drugmaker Shire has presented new data showing that switching to its Gaucher disease drug VPRIV (velaglucerase alfa for injection)) from US biotech firm Genzyme's Cerezyme (imiglucerase) is safe, in the wake of further manufacturing problems for the latter that have also caused supply shortages for its product, as reported by The Pharma Letter yesterday. 26 March 2010
Sign up to receive email updates
Join industry leaders for a daily roundup of biotech & pharma news
Today's issue
Company Spotlight
A US biotech company dedicated to creating oral drugs with an emphasis on indications in virology and immunology.
Copyright © The Pharma Letter 2024 | Headless Content Management with Blaze