Bayer's tumour-agnostic cancer drug Vitrakvi and GW's cannabis-derived Epidyolex on course for EU approval

LONDON, 26 July (APM) - Five new drugs were recommended for approval in the EU on Friday, including Bayer's Vitrakvi (larotrectinib), which would be the first tumour-agnostic cancer drug available in the region.
The European Medicines Agency (EMA)'s advisory committee CHMP backed the drug for conditional marketing authorisation for the treatment of adult and paediatric patients with solid tumours that display a neurotrophic tyrosine receptor kinase (NTRK) gene fusion, regardless of where the tumour originated.
Conditional approval means a drug can be made available based on more limited or earlier data. They are valid for one year and can be renewed annually or converted to full approval once more data are collected.
Treatment with Vitrakvi is recommended for patients whose disease has spread or cannot be surgically removed, and who have no other satisfactory treatment options, said the EMA in a statement.
The regulator described Vitrakvi as the first so-called 'histology-independent' cancer treatment recommended for approval in the EU. This means that it can be used to treat non-haematological tumours provided that the patient has the NTRK mutation as confirmed by a validated test.
The NTRK gene fusion mutation leads to the development of proteins that can cause cancer cells to grow. Vitrakvi blocks the action of these proteins, inhibiting the growth of the cancer.
The EMA said NTRK gene fusions can be observed "very frequently" in a certain number of rare cancer types that affect both adults and children.
The recommendation is based on data from a Phase III programme that included 102 adults and children who had either already received standard therapy, or would have had to undergo disfiguring surgery, or were unlikely to respond to available therapies.
For those patients who received Vitrakvi, 67% responded to the drug and of those, the response lasted six months or longer in 88% and 12 months or longer in 75%.
Vitrakvi was approved in the U.S. in November last year under the Food and Drug Administration (FDA)'s accelerated approval scheme, which also requires further data to be submitted to continue approval. The drug is also approved in Brazil and Canada and other filings are underway, said Bayer in a statement.
Bayer's senior vice president and head of oncology development Dr Scott Fields said: "Larotrectinib was specifically developed to treat patients with TRK fusion cancer, and has the potential to significantly improve treatment outcomes, regardless of tumour type or age. As researchers learn more about tumour genomics, it becomes all the more important to ensure broad access to genomic testing to allow patients that have the potential to benefit from precision medicines to be identified and treated, moving us beyond a one-size fits all therapeutic approach."
Other companies working on tumour-agnostic cancer drugs include Roche, which is developing Rozlytrek (entrectinib), another drug intended for people with NTRK mutations. It was approved in Japan last month (APMHE 63373).

GW Pharma's Epidyolex

The CHMP also gave a positive opinion to GW Pharma's Epidyolex (cannabidiol) for the treatment of seizures associated with Lennox-Gastaut syndrome or Dravet syndrome, two forms of epilepsy.
The development of the drug has gained widespread media attention as it contains an active substance derived from cannabis. It is the first drug of this kind to receive a positive opinion in the EU centralised procedure.
The EMA said in a document that the benefits with Epidyolex are its ability to help manage seizures associated with LGS and DS.
The drug was approved in the U.S. last year under the name Epidiolex (APMHE 58667).
GW's chief executive Justin Gover said in a statement: "Cannabidiol oral solution is the first in a new class of epilepsy medicines and the first plant-derived cannabis-based medicine to be submitted for European regulatory review, representing a historic breakthrough."

Inhaled levodopa for Parkinson's

The CHMP also recommended Acorda Therapeutics' Inbrija, an inhaled version of Parkinson's disease treatment levodopa.
The drug is recommended for intermittent treatment of OFF episodes in people with Parkinson's disease treated with carbidopa/levodopa. OFF episodes are periods when symptoms of Parkinson’s that result from low levels of dopamine between doses of oral carbidopa+levodopa, the standard oral baseline Parkinson’s treatment.
Levodopa has been used for decades as a treatment for Parkinson's as an oral pill. It is mainly used in combination with carbidopa, which prevents some of levodopa’s side effects. Inbrija was approved in the U.S. in December last year (APMHE 61216).

Trogarzo and generic deferasirox

Other drugs backed by the CHMP included Theratechnologies's Trogarzo (ibalizumab) for the treatment of HIV infection.
Finally, Mylan's generic version of deferasirox was recommended for the treatment of chronic iron overload due to blood transfusions in patients with beta thalassaemia major, non-transfusion-dependent thalassaemia syndromes and other anaemias.
The opinions will now go to the European Commission for final approval.



Interviews with KOLs/senior executives amongst the Regulators, Payers, Health, Medical & Pharmaceutical organisations

Events coverage with a unique focus on Market Access & sustainability of healthcare systems

6 European bureaus : Berlin, Brussels, London, Madrid, Milan & Paris

Ask for a Free trial and get access to the latest stories

Our coverage includes:
  • Health Care
  • Market Access
  • HTA – policies & practices
  • European medicine regulations
  • Drug safety issues
  • Pricing & Reimbursement
  • International medicines agencies

If you are a Payer, Pharmaceutical or Consulting professional our premium data will keep you informed on the regulatory, pricing, market access and cost-effectiveness issues that impact all stakeholders.


an initial 10 day temporary access of APM Health Europe.