Twelve medicines were recommended for approval by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) at its meeting at the end of last week.
Among them are treatments for different forms of cancer, hemophilia A, generalized myasthenia gravis and short bowel syndrome, as well as a vaccine against rabies.
The list also includes two biosimilars and two generic drugs. A positive opinion is a recommendation for approval, not a marketing authorisation for which the European Commission is responsible.
Lung cancer
Gevalka is targeted therapy for adults with advanced non-small cell lung cancer that is ALK positive.
In a study of patients receiving first-line treatment, the drug improved time without disease worsening compared to crizotinib. The selection of patients presupposes the identification of the specific molecular marker.
Platinum-resistant ovarian cancer
Lifyorli is recommended in combination with chemotherapy for adults with high-grade cancer of the ovaries, fallopian tubes or peritoneum, which is resistant to platinum.
According to the EMA, the combination improved both the time without disease getting worse and overall survival, compared to administering chemotherapy alone.
Ovarian cancer after the first chemotherapy
Sepalna is aimed at a different group of patients. It is recommended as maintenance therapy for adults with advanced high-grade ovarian, fallopian tube or peritoneal cancer who have responded fully or partially to the first platinum chemotherapy.
The drug prevents the damaged DNA of cancer cells from repairing. In the EMA's assessment, it extended the time without disease getting worse compared to placebo.
Hidodenitis suppurative
Povofortay is intended for adults with active, moderate to severe suppurative hidrodenitis, also known as reverse acne. This is a chronic inflammatory disease that can cause painful lumps, abscesses and scarring.
According to the EMA, it is the first daily oral treatment recommended for this group. In the data evaluated, it reduced skin lesions and pain compared to placebo.
Haemophilia A
Frehemgo is recommended for the prevention of bleeding events in patients with haemophilia A. The recommended indication covers all age groups, but varies depending on whether the patient has developed factor VIII inhibitors and the severity of the disease.
It is an injectable treatment that mimics the function of activated factor VIII, contributing to the clotting process. The EMA reports a reduction in bleeding episodes in the data it assessed.
Generalized myasthenia gravis
Klygefa is intended as an adjunct treatment for adults with generalised myasthenia gravis if they are positive for antibodies to the acetylcholine receptor. The disease causes muscle weakness and fatigue.
The treatment inhibits a protein of the immune system and is intended to be given weekly. A 26-week study recorded improvements in the severity of the disease, daily functions and quality of life of patients.
Short bowel syndrome: Zeydovio
Zeydovio is recommended for adults with short bowel syndrome, a condition in which the intestine cannot absorb enough fluids and nutrients, often after surgical removal of much of it.
The element noted in the EMA's assessment is the reduction of the need for parenteral support, i.e. for the administration of fluids or nutrients by infusion.
Rabies Prevention: VaxRabeo
VaxRabeo is a vaccine that is recommended for prophylaxis before or after exposure to the rabies virus in people of all ages. According to the EMA, it induces a protective antibody response in children and adults.
Biosimilars for breast cancer and allergic diseases
Pebrilzo contains pertuzumab and is intended, in combination with other treatments, for specific cases of HER2-positive breast cancer. It is a biosimilar of the already licensed Perjeta. EMA reports comparable quality, safety and efficacy. It is also the first biosimilar to be recommended for approval under the new personalized clinical assessment approach.
The second biosimilar, Adcomfo, contains omalizumab and is recommended for specific patients with severe allergic asthma, chronic rhinosinusitis with nasal polyps, or chronic spontaneous urticaria. Its reference drug is Xolair.
Finally, the committee gave a positive opinion on two generics for inflammatory and hematological diseases
Inijaq, a generic of tofacitinib, is recommended for specific forms of arthritis, ankylosing spondylitis and ulcerative colitis, as well as juvenile idiopathic arthritis under the prescribed conditions.
Ruxolitinib Zentiva, a generic of ruxolitinib, is recommended for myelofibrosis, polycythemia vera, and graft-versus-host disease, depending on the patient's characteristics and age. In both cases, these are new generic options of active substances already used in the EU
In addition, the Commission recommended an extension of the use of 11 already licensed medicines. Among them is Anzupgo, which is also recommended for use in adolescents from 12 years of age with moderate to severe chronic hand eczema.
