Regulatory Index News: 04/01/2018

Welcome to your very first 2018 Regulatory Index News update. Take a read to see the current stories of the new year.

Breakthrough status awarded to Novartis’ Kisqali

The CDK4/6 inhibitor, Kisqali, has received breakthrough status in combination with tamoxifen or an aromatase inhibitor from the FDA as an endocrine-based treatment in pre- or peri-menopausal women with hormone-receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-) advanced or metastatic breast cancer. This comes after positive data from a pre-menopausal dedicated Phase III trial, MONALEESA-7, which found that the combination Kisqali treatment prolonged progression free survival (PFS) compared to endocrine therapy alone. This will allow for an accelerated development and review of the treatment as it has the potential to substantially improve patients lives beyond that of current available therapies. Please click here for more from a PharmaTimes article.  

New FDA guidance aims for better drugmaker-reviewer communication

To start the new year off, the FDA has released two new guidance’s to improve interactions between the industry and the agency, and allow drug development to become "more informed and efficient".  One final guidance on communications, is focused on improving communication transparency and timeliness between drug developers and the FDA review staff. Another guidance is based on formal meetings with the FDA on Prescription Drug User Fee Act products, which has been created to ensure these meetings become more consistent and thus improve drug approval speed and efficiency. If you would like to read more on this, please click here for an article from BioPharmaDIVE.

GSK’s Strimvelis backed in NICE final draft guidelines

The UK’s cost assessors have published final draft guidance for GSK’s gene therapy, Strimvelis, approving it for NHS use for a rare immune deficiency condition known as severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). The condition, commonly known as bubble baby syndrome, is an inherited disease that renders the immune system dysfunctional and if left untreated, quality of life is affected resulting in early death before school age. This gene therapy uses the patient’s bone marrow cells and modifies them to allow for treatment and has now been approved to be used on the NHS when no suitably matched stem cell donor is available. Please click here to read more from a PharmaTimes article. 



Max Lymbery

Date Published

04th January 2018

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