The Type 2 diabetes medicine Trulicity (dulaglutide) has been approved in the US to treat adults with diabetes who have cardiovascular disease or are at risk of the disease. The makes Trulicity, first approved in 2014, the only Type 2 diabetes medicine that can also be used to reduce the risk of major adverse cardiovascular events.
Positive long-term data have been reported for a cell therapy being tested in patients with retinitis pigmentosa, a group of hereditary diseases of the eye that can lead to a progressive loss of vision and blindness. Results from the ongoing US Phase 1/2a study of the human retinal progenitor cell therapy showed a meaningful clinical effect at all points in time out to 12 months after treatment.
The mode of action of an experimental treatment for septic shock has been confirmed in a Phase 2 study, paving the way for further development of the therapy in patients with an early stage of the disease. Septic shock is a life-threatening organ dysfunction caused by the body’s response to infection. The trial, AdrenOSS-2, enrolled 301 patients who were identified by a novel biomarker as having early septic shock and elevated blood levels of the peptide hormone adrenomedullin. They were treated with the monoclonal antibody adrecizumab.
The US Food and Drug Administration has stepped up its oversight of certain pharmacogenetic tests that make claims for treatments that are not supported by sound science. In an announcement on 20 February, the agency said that it was introducing a new table on the internet giving a list of gene-drug interactions that are based on scientific evidence. This can be viewed at https://www.fda.gov/medical-devices/precision-medicine/table-pharmacogenetic-associations.
A new intravenous treatment for the prevention of migraine has been approved by the US Food and Drug Administration on the basis of trial data showing that patients had a sustained reduction in their disease episodes for a period of months after receiving the drug. The treatment, Vyepti (eptinezumab), is a monoclonal antibody that binds to the human calcitonin gene-related peptide with the result that migraine attacks are reduced. It was developed by Alder BioPharmaceuticals Inc, a company that was acquired by H. Lundbeck A/S of Denmark in 2019.
Galapagos ended 2019 with cash and cash equivalents of €5.8 billion, a lucrative research and development partnership with Gilead Sciences Inc, and regulatory applications in three regions for the approval of its wholly-owned product, filgotinib for rheumatoid arthritis. The company’s founder and chief executive Onno van de Stolpe said the confluence of events had ensured the company’s independence for a period of 10 years while giving it an opportunity to significantly increase the size of its portfolio.
GlaxoSmithKline Plc has expanded further into cell therapy with a partnership aimed at generating drug candidates for solid tumours. The collaboration is with Immatics Biotechnologies GmbH of Germany which has been working for several years with the MD Anderson Cancer Center in Texas, US on engineering T cells to fight cancer.
A new Belgian company has launched with €8.4 million in seed capital to develop therapies for solid tumours by investigating the interactions between perivascular macrophages and the tumour vasculature. Based on discoveries by Peter Carmeliet and Massimiliano Mazzone, the company will select the most promising targets for drug discovery and validate lead programmes for further development towards the clinic.
The first gene therapy for haemophilia to be submitted for regulatory review, valoctocogene roxaparvovec, has been given ‘priority review’status by the US Food and Drug Administration. Developed by BioMarin Pharmaceutical Inc, the therapy will be assessed for safety and efficacy for the treatment of patients with haemophilia A. Haemophilia A is the more prevalent of the two disease types, haemophilia A and B. Under a priority review, the FDA aims to issue a decision on marketing in six months rather than the usual 10, which means the outcome of the review should be known by 21 August.
UCB SA could potentially launch up to seven products by 2025 as it advances late-clinical stage programmes in epilepsy and psoriasis and looks ahead to the acquisition of Ra Pharma Inc, a US company with a product for myasthenia gravis in Phase 3 development.
The Belgian pharma gave the forecast with the release of its 2019 results on 20 February which showed revenue of €4.9 billion, up by 6% from a year earlier. The company’s core products Cimzia and Vimpat had sales of €1.7 billion and €1.3 billion respectively, both showing double-digit increases from the previous year.