Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 11-14 December 2017 Seven medicines recommended for approval, including an advanced therapy
The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommended seven medicines for approval at its December 2017 meeting, including two orphan medicines, one of which is also an advanced therapy medicinal product (ATMP).
The seven new drugs recommended for approval are:
- Alofisel (darvadstrocel), for
the treatment of complex perianal fistulas in patients
with Crohn's
disease: The CHMP recommended granting a marketing
authorisation for the ATMP Alofisel (darvadstrocel), for the
treatment of complex perianal fistulas in patients with Crohn's
disease. Alofisel has an orphan designation.
Crohn's disease is a long-term condition that causes inflammation of the digestive system or gut. Apart from affecting the lining of the bowel, inflammation may also go deeper into the bowel wall. Perianal fistulas are common complications of Crohn's disease and occur when an abnormal passageway develops between the rectum and the outside of the body. These can lead to incontinence (a lack of control over the opening of the bowels) and sepsis (blood infection). Complex fistulas are known to be more treatment resistant than simple fistulas. There is currently no cure for Crohn's disease, so the aim of treatment is to stop the inflammatory process, relieve symptoms and avoid surgery wherever possible. Crohn's disease can affect people of all ages, with a higher incidence in the younger population.
The active substance of Alofisel is darvadstrocel. Darvadstrocel contains expanded adipose stem cells which, once activated, impair proliferation of lymphocytes and reduce the release of pro-inflammatory cytokines at the inflammation sites. This immunoregulatory activity reduces inflammation and may allow the tissues around the fistula tract to heal.
The applicant for Alofisel (Darvadstrocel) is Tigenix, S.A.U.
- Crysvita
(burosumab), a
medicine for the
treatment of
X-linked hypophosphataemia: The
European Medicines Agency's (EMA) Committee for Medicinal
Products for Human Use (CHMP) has recommended granting a
conditional marketing authorisation, in the European Union, for
Crysvita (burosumab), a medicine for the treatment of X-linked
hypophosphataemia (XLH) with radiographic evidence of bone disease
in children 1 year of age and older and adolescents with growing
skeletons.
XLH is an inherited disorder characterized by low levels of phosphate in the blood. The phosphate is abnormally processed in the kidneys, which causes a loss of phosphate in the urine (phosphate wasting) and leads to soft, weak bones (rickets). In most cases, the signs and symptoms of hereditary hypophosphataemic rickets begin in early childhood. Characteristic features include bowed or bent legs, short stature, bone pain, and severe dental pain.
The CHMP recommended conditional approval for the medicine. This is one of EU's regulatory mechanisms to facilitate early access to medicines that fulfil unmet medical need. Conditional approval allows the Agency to recommend a medicine for marketing authorisation in the interest of public health where the benefit of its immediate availability to patients outweighs the risk inherent in the fact that additional data is still required. The Applicant for Crysvita (Burosumab) is Kyowa Kirin Limited.
- Ozempic
(Semaglutide) for
the treatment of
type 2 diabetes:
The Committee for Medicinal Products for Human Use (CHMP) adopted a
positive opinion, recommending the granting of a marketing
authorisation for the medicinal product Ozempic, intended for the
treatment of type 2 diabetes.
The active substance of Ozempic is semaglutide, a glucagon-like peptide 1 (GLP-1) receptor agonist. Like native GLP-1, semaglutide leads to an increase in glucose-dependent insulin secretion and a reduction in glucagon release.
Ozempic is indicated for the treatment of adults with insufficiently controlled type 2 diabetes mellitus as an adjunct to diet and exercise:
As monotherapy when metformin is considered inappropriate due to intolerance or contraindications In addition to other medicinal products for the treatment of diabetes.
The applicant for Ozempic is Novo Nordisk A/S.
- Alkindi
(Hydrocortisone) for
the treatment of
Replacement therapy
of adrenal
insufficiency in
infants, children
and adolescents: The European
Medicines Agency's (EMA) Committee for Medicinal Products for
Human Use (CHMP) has recommended granting a paediatric-use
marketing authorisation (PUMA) for Alkindi (hydrocortisone) for the
treatment of primary adrenal insufficiency, a rare hormonal
disorder, in infants, children and adolescents.
Primary adrenal insufficiency is a condition where the adrenal glands (located just above the kidneys) do not produce enough cortisol, a steroid hormone (also known as the stress hormone because it is released in response to stress). Symptoms include weight loss, muscle weakness, fatigue, low blood pressure, low blood sugar, disturbances in sodium and potassium balance and sometimes darkening of the skin. Adrenal insufficiency can be life-threatening and usually requires life-long treatment to replace the missing cortisol.
Alkindi is a paediatric-specific formulation of hydrocortisone, a glucocorticoid which is being routinely used over the last 50 years as a replacement therapy for the treatment of adrenal insufficiency in adults and children.
The applicant for Alkindi is Diurnal LTD.
- Herzuma
(Trastuzumab) for
the Treatment of
breast and
gastric cancer: The Committee for
Medicinal Products for Human Use (CHMP) adopted a positive opinion,
recommending the granting of a marketing authorisation for the
medicinal product Herzuma, intended for the treatment of breast and
gastric cancer.
Herzuma will be available as a 150mg powder concentrate for solution for infusion. The active substance of Herzuma is trastuzumab, a monoclonal antibody that binds with high affinity and specificity to HER2 leading to the inhibition of proliferation of tumour cells that overexpress HER2.
Herzuma is a biosimilar medicinal product. It is highly similar to the reference product Herceptin (trastuzumab), which was authorised in the EU on August 28, 2000.
The applicant for Herzuma (Trastuzumab) is Celltrion Healthcare Hungary Kft.
- Anagrelide
for the
treatment of
Reduction of
elevated platelet
counts in at
risk essential thrombocythaemia
patients: The Committee for Medicinal Products for
Human Use (CHMP) adopted a positive opinion, recommending the
granting of a marketing authorisation for the medicinal product
Anagrelide, intended for the reduction of elevated platelet counts
in at risk essential thrombocythaemia patients.
The active substance of Anagrelide is anagrelide, an antineoplastic agent. The precise mechanism by which anagrelidereduces bloodplatelet count is unknown. Anagrelideis aninhibitor of cyclic AMP phosphodiesterase III.
Anagrelide Mylan is a generic and hybrid of Xagrid (0.5 mg hard capsules), which has been authorised in the EU since November 16, 2004.
The full indication is: "Anagrelide is indicated for the reduction of elevated platelet counts in at risk essential thrombocythaemia (ET) patients who are intolerant to their current therapy or whose elevated platelet counts are not reduced to an acceptable level by their current therapy.
An at risk essential thrombocythaemia patient is defined by one or more of the following features:
> 60 years of age or
a platelet count > 1,000 x 109/l or
A history of thrombo-haemorrhagic events.
The applicant for Anagrelide Mylan is Mylan S.A.S.
-
Efavirenz/Emtricitabine/Tenofovir disoproxil Krka for the
Treatment of HIV infection: The Committee for Medicinal
Products for Human Use (CHMP) adopted a positive opinion,
recommending the granting of a marketing authorisation for the
medicinal product Efavirenz/Emtricitabine/Tenofovir disoproxil
Krka, intended for the treatment of HIV infection.
Efavirenz/Emtricitabine/Tenofovir disoproxil Krka contains as active substances - the antiretrovirals efavirenz, emtricitabine and tenofovir disoproxil (ATC code: J05AR06). The medicine will be available as film-coated tablets (600 mg/200 mg/245 mg). Efavirenz activity is mediated by noncompetitive inhibition of HIV reverse transcriptase, while emtricitabine and tenofovir disoproxil are substrates and competitive inhibitors of HIV reverse transcriptase. After phosphorylation, they are incorporated into the viral DNA chain, resulting in chain termination.
Efavirenz/Emtricitabine/Tenofovir disoproxil Krka is a generic of Atripla, which has been authorized in the EU since December 13, 2007.
The applicant for Efavirenz/Emtricitabine/Tenofovir disoproxil Krka is KRKA, d.d., Novo mesto.
Negative opinion on new medicinesp;sp;
The CHMP adopted a negative opinion for Aplidin (plitidepsin). Aplidin was expected to be used to treat multiple myeloma.
Three recommendations on extensions of therapeutic indication
The Committee recommended extensions of indications for
- Taltz (Ixekizumab): The CHMP adopted a new indication Psoriatic arthritis - Taltz, alone or in combination with methotrexate, is indicated for the treatment of active psoriatic arthritis in adult patients who have responded inadequately to, or who are intolerant to one or more disease modifying anti-rheumatic drug (DMARD) therapies. The marketing authorisation holder for Taltz (Ixekizumab) is Eli Lilly Nederland B.V.
- Truvada (Emtricitabine /
Tenofovir disoproxil): The CHMP adopted an extension to an
existing indication as follows -
Treatment of HIV-1 infection:
Truvada is indicated in antiretroviral combination therapy for the treatment of HIV-1 infected adults. Truvada is also indicated for the treatment of HIV-1 infected adolescents, with NRTI resistance or toxicities precluding the use of first line agents
Pre-exposure prophylaxis (PrEP):
Truvada is indicated in combination with safer sex practices for pre-exposure prophylaxis to reduce the risk of sexually acquired HIV-1 infection in adults and adolescents at high risk.
The marketing authorisation holder for Truvada (Emtricitabine / Tenofovir disoproxil) is Gilead Sciences International Limited.
- Yervoy (Ipilimumab):
The CHMP adopted an extension to the existing indication as follows
-
Yervoy is indicated for the treatment of advanced (unresectable or metastatic) melanoma in adults, and adolescents 12 years of age and older.
The marketing authorisation holder for this medicinal product is Bristol Myers Squibb Pharma EEIG.
Outcome of review on Mycophenolate
The European Medicines Agency (EMA) has updated recommendations for contraception in men and women taking mycophenolate medicines which are used to prevent rejection of transplanted organs.
Mycophenolate medicines are known to increase the risk of malformations and miscarriages during pregnancy if the fetus is exposed to them in the womb.
The CHMP has concluded that current evidence does not indicate a risk of malformations or miscarriages during pregnancy when the father has taken mycophenolate medicines (used to prevent rejection of transplanted organs), although the risk of genotoxicity cannot be completely ruled out. For male patients, the CHMP now recommends that either the male patient or his female partner use reliable contraception (it is no longer required that they both use contraception).
The updated recommendations follow a periodic review of mycophenolate medicines by EMA's Pharmacovigilance Risk Assessment Committee (PRAC), which considered the available clinical and non-clinical data.
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