Europejska Agencja Leków zatwierdziła Vitrakvi (larotrectinib) do leczenia pacjentów dorosłych oraz pacjentów pediatrycznych. Lek ukierunkowany jest na czynnik genetyczny nowotworu, a nie na lokalizację guza. Rekomendowany jest do stosowania u pacjentów z rozsianymi guzami lub w przypadkach, kiedy nowotwór nie może byc leczony chirurgicznie. Wcześniej, w grudniu 2018, lek został zatwierdzony przez FDA.
Larotrectinib wskazany jest przede wszystkim w leczeniu pacjentów z obecnością fuzji genów neurotroficznej receptorowej kinazy tyrozynowej (NTRK), z przerzutami lub gdy chirurgiczna resekcja jest niewskazana, np. z powodu zagrożenia dla pacjenta. Geny NTRK, które kodują białka TRK mogą łączyć się z innymi genami nieprawidłowo co z kolei powoduje wzrost guzów. Fuzje NTRK są rzadko spotykane, ale występują w nowotworach powstających w wielu miejscach ciała.
Zgoda została udzielona na rzecz firmy Gayer AG. Informacje EMA:
Vitrakvi is 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 (i.e. that do not begin in the blood or bone marrow) tumours with this specific mutation, regardless of where in the body the tumour originated. Before patients can be started on the medicine, the presence of the mutation in the tumour should be confirmed by a validated test.
The active substance in Vitrakvi – larotrectinib – targets a very specific genomic alteration of a patient’s tumour. This occurs when NTRK genes that encode specific proteins are abnormally fused to a gene. This mutation, called NTRK gene fusion, leads to the development of proteins that can cause cancer cells to grow. Vitrakvi blocks the action of these proteins and in doing so inhibits the growth of the cancer.
NTRK gene fusions can be observed very frequently in a certain number of rare cancer types that affect both adults and children. In addition, this gene fusion occurs rarely in some of the most common cancer types. The efficacy and safety of Vitrakvi were studied in three single-arm trials (i.e. studies with no control group) that included a total of 102 adults and children with cancer that were evaluated. These patients had either already received standard therapy, or would have had to undergo disfiguring surgery, or were unlikely to respond to available therapies.
The share of patients who responded to treatment with Vitrakvi was 67%. Of those, the response lasted six months or longer in 88% and 12 months or longer in 75%. Tumour responses were seen both in rare tumour types such as infantile fibrosarcoma and salivary gland tumours, as well as in common diseases such as lung and colon cancer.
The most common side effects were tiredness, increased levels of liver enzymes, dizziness, constipation, nausea, anaemia (low red blood cell count), and vomiting. The CHMP recommended a conditional approval for this medicine. This is one of the EU’s regulatory mechanisms to facilitate early access to medicines that fulfil an unmet medical need. This type of approval allows the Agency to recommend a medicine for marketing authorisation with less complete data than normally expected, in cases where the benefit of a medicine’s immediate availability to patients outweighs the risk inherent in the fact that not all the data are yet available.
/ mzdrowie