News

A new gene therapy has been approved in Europe, paving the way for its use in patients with beta-thalassaemia, a rare blood disorder. The treatment, Zynteglo, uses a lentiviral vector to deliver functional copies of a beta-globin gene into a patient’s haematopoietic stem cells in order to correct for a shortage of beta-globin. It was given a positive opinion by the European Medicines Agency on 29 March and a conditional approval by the European Commission on 29 May.

The US Food and Drug Administration has approved an antibody-drug conjugate from Roche for diffuse large B cell lymphoma (DLBCL), an aggressive cancer that grows quickly in the lymph nodes and may affect the bone marrow, spleen, liver or other organs. The drug targets CD79b, a protein expressed on B cells.

Olivier Brandicourt is to retire as chief executive of Sanofi SA and be replaced by 51-year-old Paul Hudson, head of Novartis Pharmaceuticals. Mr Hudson will take up his position on 1 September marking the third change in Sanofi’s top leadership in a little more than a year.

Evotec SE has expanded a partnership with Celgene Corp to identify new treatments for neurodegenerative diseases using induced pluripotent stem (iPS) cells. This has triggering a milestone payment to the German company of $9 million.

Genmab A/S, one of Europe’s oldest antibody companies, has registered to make a public offering of American Depositary Shares on the US Nasdaq market of up to $500 million. The plans were disclosed on 28 May but as of 6 June the final size of the offering had not been decided. The company is currently listed on Nasdaq Copenhagen and is proposing the US listing to broaden its shareholder base.

Denmark-based Abacus Medicine A/S announced on 29 May that it was postponing an initial public offering planned for the Frankfurt Stock Exchange because of unfavourable market conditions. The parallel trade company was reportedly planning to raise €50 million to finance expansion.

The company’s biggest market is Germany where it sells drugs purchased at cheaper prices in other European markets.

It is estimated that up to 7% of patients with pancreatic cancer have a mutation in the tumour suppressor genes BRCA1 and BRCA2, impairing the body's ability to repair damaged DNA and allowing tumours to grow. BRCA mutations also drive ovarian and breast cancers, but they can be particularly lethal in pancreatic tumours because this cancer is difficult to detect at an early stage.

Novo Holdings A/S is to make an equity investment of up to £53.5 million in Oxford Biomedica Plc which has grown a business around the supply of lentiviral vectors for third parties including Novartis, the developer of Kymriah, the first gene therapy for cancer to be approved by the US Food and Drug Administration. Following the transaction Novo will have a stake of up to 10.1% in the UK company.

Novartis has decided to price its new gene therapy for spinal muscular atrophy using a formula that measures the performance of the drug over five years. The therapy, Zolgensma (onasemnogene abeparvovec-xioi), is a one-time treatment for a genetic disease that affects a child’s ability to swallow and breath, frequently leading to death.

The US Food and Drug Administration has approved a gene therapy to treat children under the age of two years who have spinal muscular atrophy, a rare genetic disease that affects a child’s ability to swallow and breath, frequently leading to death.