Regulatory

Woman having epileptic fit

Treatment for severe form of epilepsy approved in Europe

21 December 2022

UCB’s Fintepla (fenfluramine) oral solution has been recommended for marketing authorisation in the European Union (EU) for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), a rare, severe form of epilepsy.  The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency based its positive opinion on safety and efficacy data […]

Gene therapy for haemophilia

First gene therapy for haemophilia B gets go ahead in Europe

20 December 2022

The first gene therapy for haemophilia B could soon be available to patients in Europe following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP).  The CHMP has recommended conditional marketing authorisation of CSL’s etranacogene dezaparvovec, a one-time gene therapy for the treatment of appropriate adults with the disease.  The therapy […]

MHRA

UK licence extension approved for AstraZeneca’s dapagliflozin

14 December 2022

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted a licence extension for dapagliflozin for the treatment of symptomatic chronic heart failure (HF).  The drug is approved in patients with HF and a left ventricular ejection fraction (LVEF) >40%, including HF with mildly reduced ejection fraction (HFmrEF) and preserved ejection fraction (HFpEF).  HF […]

Drug development practices to improve public health policy 

6 December 2022

Dr Andrew Garrett, Executive Vice President of Scientific Operations at ICON, explores the benefits of integrating some of the validation and standardisation practices already developed for PIs into the practices around NPIs and public health data; outlines some of the practices that can be implemented in order to do this; and highlights the need for […]

Hemp plants being used in scientific research

UK approval for CBD pain treatment

2 December 2022

The UK Medicines and Healthcare Products Regulatory Agency (MHRA) has approved a CBD/THC combination treatment under its Named Patient programme and other compassionate areas. Medlab’s NanaBis, to be used under NanaDol, a botanical version of NanaBis, has previously shown encouraging Phase I/II data for the treatment of cancer-induced bone pain and could be a potential […]

Dystrophin muscle protein

Gene therapy granted FDA Priority Review

2 December 2022

The US Food and Drug Administration (FDA) has accepted Sarepta Therapeutics’ Biologics License Application (BLA) seeking accelerated approval of SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulant individuals with Duchenne muscular dystrophy.   The therapy, which is being developed in partnership with Roche, has been granted Priority Review by the FDA, with a regulatory action date […]

Prostate cancer awareness

UK fast tracks approval for life-extending prostate cancer drug

29 November 2022

England will become the first country in Europe to roll out darolutamide (Nubeqa) to patients whose prostate cancer has spread to other parts of the body.  The drug works by blocking androgen receptors in cancer cells, which in turn blocks the effect of testosterone that allows the cancer cells to survive and multiply. Studies show […]

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FDA approves first gene therapy for haemophilia B

24 November 2022

The US Food and Drug Administration (FDA) has approved Hemgenix (etranacogene dezaparvovec-drlb), the first and only one-time gene therapy for appropriate adults with haemophilia B.   The drug is approved for the treatment of adults with haemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening haemorrhage or have repeated, serious […]

Patient with type 1 diabetes

FDA approves first disease-modifying therapy for T1D

21 November 2022

The US Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for anti-CD3-directed antibody TZIELD (teplizumab-mzwv), the first immunomodulatory treatment for type 1 diabetes (T1D).   It is approved for intravenous use to delay the onset of stage 3 T1D in adult and paediatric patients aged eight years and older with stage 2 […]

Child having physiotherapy

Gene therapy could ‘transform prognosis’ for AADC deficiency patients 

21 November 2022

PTC Therapeutics’ Upstaza (eladocagene exuparvovec) has received authorisation from the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK.   Upstaza is the first approved disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency. It is also the first marketed gene therapy directly infused into the brain. The MHRA marketing authorisation is for treatment of […]

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