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Biotechnology
Shares of bluebird bio closed down 18% at $10.03 yesterday, after it announced that the FDA has placed its clinical program for lovotibeglogene autotemcel (lovo-cel) - previously known as LentiGlobin - as a gene therapy for sickle cell disease (SCD) on partial clinical hold for patients under the age of 18. 21 December 2021
Pharmaceutical
The US Food and Drug Administration has granted accelerated approval for Oxbryta (voxelotor), from USA-based Global Blood Therapeutics, to treat sickle cell disease in pediatric patients aged four up to 11 years. 20 December 2021
Generics
Swiss drugmaker Vifor Pharma and its partner American Regent, Daiichi Sankyo’s US subsidiary, today announced that they have reached settlement agreements with Mylan Laboratories, part of Viatris, the newly-formed generics power-house resulting from the merger of Mylan and Pfizer, and Sandoz, which is about to be spun out of Novartis, that resolve the patent litigation brought in response to Abbreviated New Drug Applications seeking approval by the US Food and Drug Administration to market a generic version of Injectafer (ferric carboxymaltose). 20 December 2021
Pharmaceutical
On Friday, the European Medicines Agency’s human medicines committee (CHMP) announced recommendations of novel medicines for approval at its December 2021 meeting. 18 December 2021
Biotechnology
Gilead Sciences subsidiary Kite and Japanese drugmaker Daiichi Sankyo yesterday announced that Yescarta (axicabtagene ciloleucel), a chimeric antigen receptor (CAR) T-cell therapy, will be available to patients with relapsed or refractory large B-cell lymphomas in Japan through the first treatment center now authorized by Daiichi Sankyo. 17 December 2021
Biotechnology
The US Food and Drug Administration (FDA) has approved Orencia (abatacept) alongside a calcineurin inhibitor and methotrexate to prevent acute graft versus host disease (aGvHD) in patients undergoing hematopoietic stem cell transplant from a matched or 1 allele-mismatched unrelated donor. 16 December 2021
Pharmaceutical
After a few of days speculation and rumors, Australia-headquartered biotech CSL Limited today confirmed it has entered into a definitive agreement to acquire Switzerland-based Vifor Pharma, a specialty pharmaceutical company with leadership in iron deficiency, nephrology and cardio-renal therapies. 14 December 2021
Biotechnology
Long-suffering investors in bluebird bio had a rare reason for cheer as the company presented positive results for betibeglogene autotemcel (beti-cel) at the American Society of Hematology (ASH) Annual Meeting. 13 December 2021
Biotechnology
US pharma major Bristol Myers Squibb has announced the first disclosure of results from a prespecified interim analysis of the pivotal TRANSFORM study at the American Society of Hematology (ASH) Annual Meeting. 13 December 2021
Biotechnology
Updated results from the ASCEND Phase III trial showed that Calquence (acalabrutinib) maintained a statistically-significant progression-free survival (PFS) benefit at three years compared to investigator’s choice of rituximab combined with either idelalisib (IdR) or bendamustine (BR) in adults with relapsed or refractory chronic lymphocytic leukemia (CLL), the most common type of leukemia in adults, said the drug’s developer, Anglo-Swedish pharma major AstraZeneca 13 December 2021
Pharmaceutical
At the 63rd annual meeting of the American Society of Hematology (ASH), Swiss pharma giant Novartis has announced new positive data for Scemblix (asciminib). 13 December 2021
Pharmaceutical
Virtually every drugmaker with a presence in hematology is preparing for their end-of-year attempt to impress physicians and investors with the best of their freshest data at the American Society of Hematology (ASH) Annual Meeting and Exposition. 10 December 2021
Biotechnology
Dutch gene therapy company uniQure and CSL Behring, part of Australia’s CSL Limited, have announced encouraging results on etranacogene dezaparvovec. 9 December 2021
Biotechnology
The UK's National Institute for Health and Care Excellence (NICE) has issued the Final Appraisal Decision (FAD) recommending the use of Aspaveli (pegcetacoplan) in adults with paroxysmal nocturnal hemoglobinuria (PNH) in England and Wales. 9 December 2021
Pharmaceutical
The Russian government will allocate about 62 billion roubles ($840 million) in 2022 for the procurements of high-priced drugs as part of the 14 high-cost nosologies (14 VZN) program, a state program, which involves procurements of the most expensive drugs for state needs in Russia, reports The Pharma Letter’s local correspondent. 7 December 2021
Pharmaceutical
Swiss pharma giant Novartis is confident that its sales total will grow by at least 4% at a compound annual growth rate (CAGR) through 2026, investors were told at an R&D day Thursday. 3 December 2021
Biotechnology
A new combo therapy has been approved by the US Food and Drug Administration, unlocking another option for people with multiple myeloma. 2 December 2021
Biotechnology
Last week’s notable news included US biotech BioMarin Pharmaceuticals receiving Food and Drug Administration approval for its Voxzogo in the treatment of achondroplasia, the most common form of dwarfism. Arrowhead Pharmaceuticals signed a heavily back-loaded agreement with the UK’s GlaxoSmithKline for its non-alcoholic steatohepatitis (NASH) candidate ARO-HSD, marking the British firm’s first foray into the sector that has seen many disappointments. Among regulatory developments, Kura Oncology had a setback last Tuesday, when the FDA put a partial clinical hold on its acute myeloid leukemia (AML) trial of KO-539 following a patient death. Finally, Ionis and partner Pfizer released Phase IIb clinical trial results for their cholesterol lowerer vupanorsen that, while meeting its primary endpoint, raised a number of questions. 28 November 2021
Biotechnology
The UK subsidiary of Italian family-owned Chiesi Farmaceutici announced yesterday that pegunigalsidase alfa (PRX-102) was awarded Innovation Passport status by the UK Medicines and Healthcare products Regulatory Agency (MHRA) on August 25, 021. 26 November 2021
Biotechnology
Fitusiran, an investigational siRNA therapeutic targeting antithrombin for the treatment of hemophilia, has shown its efficacy in late-stage trials. 25 November 2021
Biotechnology
US healthcare giant Johnson & Johnson today revealed it has submitted applications with the US Food and drug Administration (FDA) and European Medicines Agency (EMA) to expand the approved indications for its Darzalex (daratumumab) subcutaneous (SC) formulation for the treatment of high-risk smouldering multiple myeloma (SMM). 8 November 2024
Biotechnology
UK health technology assessor the National Institute for Health and Care Excellence (NICE) issued final guidance recommending reimbursement of Ayvakyt (avapritinib) from USA-based Blueprint Medicines. 6 November 2024
Biotechnology
French drugmaker Sanofi has revealed the results from the Phase III LUNA 3 trial of rilzabrutinib, an oral bruton tyrosine kinase (BTK) inhibitor, in persistent or chronic immune thrombocytopenia (ITP). 6 November 2024
Biotechnology
Beam Therapeutics will detail new clinical and preclinical data on its sickle cell disease (SCD) therapies at the upcoming American Society of Hematology (ASH) meeting. 6 November 2024
Pharmaceutical
An impressive set of quarterly financial results and an improved outlook for the rest of the year were announced by Bristol Myers Squibb on Thursday. 31 October 2024
Biotechnology
Maryland, USA–based glycobiology specialist GlycoMimetics has entered into an acquisition agreement with Crescent Biopharma, a privately held biotechnology company advancing a pipeline of oncology therapeutics designed to treat solid tumors. 30 October 2024
Pharmaceutical
Novartis late yesterday revealed that Scemblix (asciminib) was granted accelerated approval by the US Food and Drug Administration (FDA) for adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). 30 October 2024
Biotechnology
Last week Vertex Pharmaceuticals released positive Phase III results for its suzetrigine for moderate-to-severed pain. Belgium’s UCB revealed that Swiss pharma giant Roche was terminating its collaboration on the Alzheimer’s candidate bepranemab. The UK’s health technology assessor the Institute for Health and Clinical Excellence (NICE) declined to recommend a second Alzheimer’s treatment, this time Eli Lilly’s Kisunla. Also of note, Lyell Immunopharma announced its planned acquisition of ImmPACT Bio and reprioritization of its own clinical pipeline. 27 October 2024
Biotechnology
California, USA-based Lyell Immunopharma (Nasdaq: LYEL) has entered into a definitive agreement to acquire privately-held US CAR-T company ImmPACT Bio. 26 October 2024
Pharmaceutical
Sanofi reported 15.7% sales growth for the third quarter of the year, reaching 13.4 billion euros ($14.2 billion), with adjusted earnings per share (EPS) rising by 12.2% to 2.86 euros, surpassing many forecasts. 25 October 2024
Biotechnology
Johnson & Johnson has secured European approval for an expanded indication of Darzalex (daratumumab) in a subcutaneous formulation. 24 October 2024
Biotechnology
Swedish Orphan Biovitrum, the Nordic biotech company also known as Sobi, today announced significantly improved financial results for the third quarter of 2024, sending the firm’s shares up 2.5% to 333.60 kronor in early trading. 24 October 2024
Biotechnology
US clinical-stage biotech AvenCell Therapeutics announced that it has raised $112 million in Series B financing, which was led by global life sciences investor Novo Holdings. 23 October 2024
Pharmaceutical
Swiss pharma giant Roche this morning reported that, in the first nine months of 2024 group sales increased by 6% at CER (2% in francs) to 45.0 billion francs ($52.0 billion). 23 October 2024
Pharmaceutical
UK pharma major AstraZeneca’s Alexion Rare Disease unit has received a positive recommendation for Voydeya (danicopan), from the UK’s National Institute for Health and Care Excellence (NICE). 22 October 2024
Biotechnology
The European Medicines Agency’s (EMA) human health committee, CHMP, announced six positive opinions of novel medicines, as well as two biosimilars, following its October meeting. 18 October 2024
Biotechnology
The US Food and Drug Administration (FDA) on Friday approved US pharma giant Pfizer’s Hympavzi (marstacimab-hncq). 14 October 2024
Biotechnology
US healthcare giant Johnson & Johnson’s Tecvayli (teclistamab) has been recommended by the National Institute for Health and Care Excellence (NICE) as a first-line option to treat myeloma in England and Wales. 10 October 2024
Biotechnology
The US Food and Drug Administration (FDA) has granted the Gilead Sciences subsidiary Kite a Regenerative Medicine Advanced Therapy Designation (RMAT) for Yescarta (axicabtagene ciloleucel) for adults with newly-diagnosed, high-risk large B-cell lymphoma (LBCL). 3 October 2024
Pharmaceutical
AstraZeneca’s supplemental New Drug Application (sNDA) for Calquence (acalabrutinib) has been accepted and granted Priority Review in the USA for the treatment of adults with previously untreated mantle cell lymphoma (MCL). 3 October 2024
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