- Global Pharma News & Resources

MHRA approves licence extension for Novartis’ targeted therapy for advanced breast cancer patients in Great Britain

  • Piqray®(alpelisib), in combination with fulvestrant is approved for appropriate advanced breast cancer patients with a PIK3CA mutation after disease progression following endocrine-based therapy, providing eligible patients with more treatment options
  • This treatment option represents more than 20 years of research and development into the role of PIK3CA mutations in breast cancer

 Today Novartis announced that the Medicines and Healthcare products Regulatory Agency (MHRA) has approved the marketing authorisation to extend the licence in Great Britain for Piqray® (alpelisib) for use in combination with fulvestrant for the treatment of postmenopausal women, and men, with hormone receptor positive(HR+), human epidermal growth factor receptor 2 negative(HER2-), locally advanced or metastatic breast cancer with a PIK3CA mutation after disease progression following endocrine-based therapy.

Resistance to endocrine therapy is a significant concern in the treatment of advanced breast cancer. PIK3CA mutations are a driver of developing endocrine resistance,[i] which may lead to faster disease progression for patients, and a worse prognosis.[ii] Endocrine resistance affects approximately 40% of HR+/HER2- advanced breast cancer patients.[iii] There is a need for new therapies that are able to specifically target the PIK3CA mutation, therefore overcoming endocrine resistance; with the goal of improved efficacy with a manageable toxicity profile that may maintain quality of life.[iv]

Dr Marina Parton, consultant oncologist in Breast Cancer at The Royal Marsden explains, “This approval is a significant development in advanced breast cancer, providing patients with additional treatment options; where there is much need for innovation that provides better clinical and patient outcomes. Targeted therapies such as alpelisib for HR+/HER2- advanced breast cancer with the PIK3CA mutation, are a step in the right direction to improve the standard of care and enable more eligible patients in Great Britain to gain access to the best possible treatments available.”

In July 2020, the European Medicines Agency (EMA) approved alpelisib for use in combination with fulvestrant for advanced breast cancer patients with a PIK3CA mutation after disease progression following endocrine therapy as monotherapy.[v]

The EMA licence did not include patients that have received prior endocrine and CDK4/6 inhibitor combination treatment, therefore to ensure greater access for all eligible patients in Great Britain, Novartis decided to proceed with the national approval process and submitted to the MHRA for a broader licence. 

Alpelisib in combination with fulvestrant has demonstrated consistent, significant improvements in median progression-free survival and overall response rates in endocrine resistant HR+/HER2- advanced breast cancer with a PIK3CA mutation, and has a generally well-tolerated and manageable safety profile when administered with appropriate and timely patient care and follow up, regardless of line of therapy or prior treatment.[vi]

In the UK, breast cancer is the most common type of cancer, with around 55,000 women diagnosed with breast cancer each year.[vii],[viii] Approximately 30% of women with earlier stages of breast cancer will develop advanced disease.[ix] Advanced breast cancer is an incurable disease, it is known that only 66% of women will survive for over a year or more and around 26.2% for five years when diagnosed at the latest stage.[x],[xi]

Heather Moses, Country Medical Director at Novartis Oncology UK says, “An advanced breast cancer diagnosis is devastating for patients, their families and their loved ones, which is why it is our priority to reimagine how we deliver care for these people. We wanted to ensure that all eligible patients in Great Britain have access to this treatment option by securing a broader licence through the MHRA. This marketing authorisation not only demonstrates our bold patient-first approach and ongoing commitment to the breast cancer community, but continued alignment to the priorities for the NHS in its Long-Term Plan for cancer patients. Despite the challenges of the COVID-19 pandemic, we have continued to partner with the NHS and the entire health ecosystem in order to identify new pathways or mutations, such as PIK3CA, that may play a role in disease progression, and identify and implement solutions for these patients with the aim of improving their quality of life.”

As of July 2021, alpelisib is now approved in 64 countries, including the US and EU member states, with regulatory submissions under review in several additional markets. The approved European indication applies to all 27 EU member states plus Northern Ireland (UK), Iceland, Norway and Liechtenstein.

Novartis aims to see that its innovative solutions will continue to help address some of society’s biggest healthcare challenges for patients today and tomorrow; driving forward genuine advances, striving for cure, especially in breast cancer. Novartis’ collaborative approach, working across the entire health ecosystem, provides us with an exciting opportunity to bring about real change to patients in Great Britain and support our pipeline.

Novartis has over 30 years of heritage in oncology, spanning back to the approval of the first targeted therapy and beyond, transforming the lives of people living with cancer and life-threatening blood disorders. Novartis’ bold ambition is grounded in its industry-leading oncology and haematology pipeline of more than 45 different compounds across 70+ development programmes. Novartis is at the forefront of medicines development. It has been the number one commercial sponsor of UK clinical trials over the past ten years and believe it is their responsibility to continue to move at pace and deliver innovations that are laser-focused on unmet patient need and the requirements of health systems.



[i] Miller TW, Hennessy BT, Gonzalez-Angulo AM, et al. Hyperactivation of phosphatidylinositol-3 kinase promotes escape from hormone dependence in estrogen receptor-positive human breast cancer. J Clin Invest. 2010;120(7):2406-2413.

[ii] Sobhani N, Roviello G, Corona SP et al. The prognostic value of PI3K mutational status in breast cancer: a meta-analysis. J Cell Biochem. 2018;119(6):4287-4292.

[iii] Sabine VS_J Clin Onocol_pg2951-2958_2014.

[iv] Mosele F et al. Outcome and molecular landscape of patients with PIK3CA-mutated metastatic breast cancer. Annals of Oncology, Volume 31, Issue 3, 377 – 386.

[v] Piqray SmPC. EMA. Available at : [Accessed December 2021]

[vi] André F, Ciruelos E, Rubovszky G. Alpelisib for PIK3CA-Mutated, Hormone-Receptor-Positive Advanced Breast Cancer. N Eng J Med. 2019.

[vii] NHS Choices (2019). Overview - Breast cancer in women. NHS. Available from: [Accessed December 2021].

[viii] Cancer Research UK. Breast cancer incidence (invasive) statistics. Available from: [Accessed December 2021].

[ix] O'Shaughnessy J. Extending survival with chemotherapy in metastatic breast cancer. The Oncologist. October 2005, 10(suppl.): 20-29.

[x] Cancer Research UK. Breast cancer survival statistics. Available from: [Accessed December 2021]  

[xi] ONS. Cancer survival in England – adults diagnosed 2013-2017. [Accessed December 2021]

UK | DECEMBER 2021 | 162255

Editor Details

Last Updated: 24-Dec-2021