News

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.

AstraZeneca Plc returned to growth in 2019 with product income of $23.6 billion, up by 12% from a year earlier, and nine drugs achieving sales of $1 billion or more. Pascal Soriot, the chief executive, predicted that 2020 would be another year of progress as new molecules complete clinical development and enter registration.

New confirmed cases of infections from the coronavirus in China, also known as COVID-19, are “generally in decline,” China’s ambassador to the EU, Zhang Ming, told journalists on Tuesday. The ambassador was speaking at a meeting in Brussels, organised by the think tank EU-Asia. Topics included EU-China trade relations, but the virus epidemic dominated the conversation. Mr Ming was at pains to detail China’s efforts to contain the epidemic and predicted that the contagion would soon be overcome.

Pheno Therapeutics Ltd has been launched in the UK with Series A funding of £5 million to develop new treatments for multiple sclerosis that would halt progression of the disease by targeting the nervous system. Current therapies mainly focus on modulating the immune system in order to reduce the severity and frequency of relapses.

OMass Therapeutics Ltd, an Oxford University spin-out that is using mass spectrometry to discover new drugs, has raised an additional £27.5 million in an extended Series A financing, bringing total proceeds from the round up to £41.5 million. The new capital will be used to progress a pipeline of small molecule therapeutics for patients with immunological and genetic disorders, enabling the company to bring its lead programme into preclinical development. The latest funding was supplied by the existing investors Syncona Ltd and Oxford Sciences Innovation.

H. Lundbeck A/S saw revenue decline in 2019 as generic competition took sales away from Onfi, its legacy treatment for Lennox-Gastaut syndrome. However, four of the company’s newer products generated sales of DKK 9.4 billion, or just over half of group revenue for the year. Total group revenue was DKK 17 billion (€2.28 billion), down by 6% from a year earlier. Profit before tax was DKK 3.5 billion, down by 34%. Driving the profit lower were amortisations of DKK 854 million and acquisition and integration costs of DKK 514 million.

AurorA Science, a holding company created to support innovation in the life sciences, including gene therapy, has been launched in Milan, Italy with €10 million in capital. The investors are Rottapharm Biotech and Italfarmaco, two companies with an established presence in the sector. The investors have the means to increase their commitments in the future.

Two experimental drugs that were being tested in people with dominantly inherited Alzheimer’s disease failed to meet their primary endpoint in a study sponsored by  Washington University School of Medicine in St. Louis, US.

Called DIAN-TU, the study was designed to find out whether either treatment could slow the rate of cognitive decline and improve disease-related biomarkers in people known to have a genetic mutation for inherited Alzheimer’s disease.

Sanofi SA reported sales of €36 billion for 2019, up by 4.8% from a year earlier but net income plunged by 34.8% to €2.8 billion following a write-down of €3.6 billion largely due to weak sales of Eloctate, a product for haemophilia A. Eloctate and Alprolix, a product for haemophilia B, entered Sanofi’s portfolio in 2018 when it bought the Biogen Inc spin-out  Bioverativ Inc. Both products are factor replacement therapies which are being challenged by new therapeutic modalities.