Vijoice

Application withdrawn

The application for this medicine has been withdrawn

alpelisib

Overview

On 30 October 2023, Novartis withdrew its application for a marketing authorisation of Vijoice for the treatment of PIK3CA-related overgrowth spectrum (PROS), a genetic condition that causes a range of symptoms, including malformations and abnormal growth or tumours affecting several tissues, such as the skin, bones, blood vessels and brain.

What is Vijoice and what was it intended to be used for?

Vijoice was developed as a medicine for treating adults and children from 2 years of age with severe or life-threatening symptoms of PROS requiring systemic therapy (therapy that affects the whole body).

Vijoice contains the active substance alpelisib and was to be available as tablets to be taken by mouth.

Vijoice was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 26 March 2021 for the treatment of PROS. Further information on the orphan designation can be found on the Agency’s website.

How does Vijoice work?

In patients with PROS, mutations (changes) in a gene called PIK3CA lead to the activation of an enzyme (PI3 kinase) that is involved in cell growth. This results in the abnormal growth of tissues (tumours) and malformations. 

The active substance in Vijoice, alpelisib, blocks the action of the enzyme and was expected to reduce the symptoms of PROS.

What did the company present to support its application?

The company presented data from a study involving 18 adults and 39 children and adolescents with PROS who were receiving Vijoice as part of a compassionate use programme. The patients had severe or life-threatening symptoms of PROS requiring systemic therapy. The main measure of effectiveness was the number of patients who, after 24 weeks of treatment, had a reduction of at least 20% in the total size of one to three abnormal growths or tumours. 

How far into the evaluation was the application when it was withdrawn?

The application was withdrawn after the European Medicines Agency had evaluated the information from the company and prepared questions. The company had not responded to the last round of questions at the time of the withdrawal.

What did the Agency recommend at that time?

Based on the review of the data and the company’s response to the initial Agency’s questions, at the time of the withdrawal, the Agency had some concerns and its provisional opinion was that Vijoice could not have been authorised for the treatment of PROS.

The Agency considered that the data from the study were not sufficient to show exactly what effect the medicine had on the size of the tumours and whether patients actually benefited from any reduction in tumour size. Furthermore, although PROS includes different types of syndromes, an effect of Vijoice was only seen for one of these syndromes. Finally, the long-term safety of the medicine, in particular its effects on growth and development in children, is unknown.

Therefore, at the time of the withdrawal, the Agency’s opinion was that the company had not provided enough data to support the application.

What were the reasons given by the company for withdrawing the application?

In its Withdrawal letter: Vijoice notifying the Agency of the withdrawal of the application, the company stated that it needed more time to obtain further data to support the evaluation of Vijoice.

Does this withdrawal affect patients in clinical trials or compassionate use programmes?

The company informed the Agency that the withdrawal has no consequences for patients in clinical trials or in compassionate use / managed access programmes using Vijoice.

If you are in a clinical trial or a compassionate use / managed access programme and need more information about your treatment, speak with your clinical trial doctor. 

Questions and answers on the withdrawal of application for the marketing authorisation of Vijoice (alpelisib)

Reference Number: EMA/492147/2023

Key facts

• Name of medicine:
  Vijoice
• Active substance:
  Alpelisib
• International non-proprietary name (INN) or common name:
  alpelisib
• Therapeutic area (MeSH):
  • Genetic Diseases, Inborn
  • Growth Disorders
• Anatomical therapeutic chemical (ATC) code:
  L01EM03
• EMA product number:
  EMEA/H/C/005468
• Orphan:
  <p>This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see <a href="/node/69983">Orphan designation</a>.</p>
• Marketing authorisation applicant:
  Novartis Europharm Limited
• Withdrawal of application:
  30/10/2023

All Documents

Withdrawal letter: Vijoice

Withdrawal assessment report : Vijoice

AdoptedReference Number: EMA/506300/2023