The European Medicines Agency's Committee for Medicinal Products for Human Use has adopted a negative opinion for Pfizer's Elelyso, while recommending Cuprymina, Revestive, Enurev Breezhaler, and Zinforo for marketing in the EU.
IHS Global Insight Perspective | |
Significance | The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has issued a negative opinion for US pharma Pfizer's Elelyso (taliglucerase alpha) not recommending it to the European Commission for marketing authorisation. |
Implications | The CHMP further adopted positive recommendations for four drugs, and a positive scientific opinion for a vaccine—Hexaxim—to be used outside the EU. |
Outlook | The recommendations by the CHMP (except in the case of Hexaxim) will be sent to the European Commission, which is expected to issue a final marketing authorisation decision within a few weeks. |
The European Medicines Agency's (EMA's) Committee for Medicinal Products for Human Use (CHMP) has not recommended United States pharma Pfizer's Elelyso (taliglucerase alpha), indicated in the treatment of type 1 Gaucher's disease, for marketing authorisation in the EU. The opinion was adopted at the committee's June meeting. At the same meeting, positive recommendations for the marketing of four drugs were issued, as well as a positive recommendation for a drug to be used outside the EU.
Positive Recommendations for New Drugs | ||
Drug | Company/Organisation | Indication |
Cuprymina (copper [64cu] chloride) | Sparkle (Italy) | Cuprymina, a radiopharmaceutical precursor, is not intended for direct use in patients. It is indicted only for the radiolabelling of carrier molecules that have been specifically developed and authorised for radiolabelling with this radionuclide. |
Revestive (teduglutide) | Nycomed (Switzerland) | Treatment of adult patients with Short Bowel Syndrome. Patients should be stable following a period of intestinal adaptation after surgery. |
Enurev Breezhaler/Seebri Breezhaler/Tovanor Breezhaler (glycopyrronium bromide) | Novartis (Switzerland) | Maintenance bronchodilator treatment to relieve symptoms in adult patients with chronic obstructive pulmonary disease. |
Zinforo (ceftaroline fosamil) | AstraZeneca (UK) | Indicated in adults for the treatment of complicated skin and soft tissue infections, and of community-acquired pneumonia. It is further stated that consideration should be given to official guidance on the appropriate use of antibacterial agents. |
Positive Recommendations for New Drugs to Be Used Outside EU | ||
Drug | Company/Organisation | Indication |
Hexaxim (diphtheria, tetanus, pertussis, hepatitis B, poliomyelitis, and haemophilus influenzae type B conjugate vaccine) | Sanofi (France) | Primary and booster vaccination of infants and toddlers from 6 weeks to 24 months of age against diphtheria, tetanus, pertussis, hepatitis B, poliomyelitis (polio) and invasive diseases caused by Haemophilus influenzae type b. |
The CHMP further issued positive recommendations for extending the therapeutic indications for three drugs. These were Afinitor (everolimus), currently approved in indications for the treatment of neuroendocrine tumours of pancreatic origin and the treatment of renal cell carcinoma; Enbrel (etanercept), currently approved in juvenile idiopathic arthritis indications; and Humira (adalimumab), currently indicated for the treatment of adults with severe active ankylosing spondylitis who have had an inadequate response to conventional therapy. The committee also issued an amendment in the indication for Cayston (aztreonam), which is highlighted below.
Positive Opinions for Extensions | ||
Drug | Company/Organisation | Additional Indication |
Afinitor (everolimus) | Novartis (Switzerland) | Indicated for the treatment of hormone receptor-positive, HER2-negative advanced breast cancer, in combination with exemestane, in post-menopausal women without symptomatic visceral disease after recurrence or progression following a non-steroidal aromatase inhibitor. |
Cayston (aztreonam) | Gilead (US) | Suppressive therapy of chronic pulmonary infections due to Pseudomonas aeruginosa in patients with cystic fibrosis (CF) aged six years and older. Consideration should be given to official guidance on the appropriate use of antibacterial agents. |
Enbrel (etanercept) | Pfizer (US) | Treatment of polyarthritis (rheumatoid factor positive or negative) and extended oligoarthritis in children and adolescents from the age of two years who have had an inadequate response to or have proved intolerant to methotrexate. Treatment of psoriatic arthritis in adolescents from the age of 12 years who have had an inadequate response to or who have proved intolerant to methotrexate. Treatment of enthesitis-related arthritis in adolescents from the age of 12 years who have had an inadequate response to or have proved intolerant to conventional therapy. |
Humira (adalimumab) | Abbott (US) | The treatment of adults with severe axial spondyloarthritis (AS) without radiographic evidence of AS but with objective signs of inflammation by elevated C-reactive protein and/or magnetic resonance imaging, who have had an inadequate response to or are intolerant to non-steroidal anti-inflammatory drugs. |
The CHMP also issued positive opinions for two generic drugs. These were US firm Mylan's Zoledronic acid (zoledronic acid), a copy of Swiss pharma Novartis's Zometa, indicated in the prevention of skeletal related events (pathological fractures, spinal compression, radiation or surgery to bone, or tumour-induced hypercalcaemia) in adult patients with advanced malignancies involving bone, and the treatment of adult patients with tumour-induced hypercalcaemia; and Swedish firm Meda's Zyclara (imiquimod), a hybrid of Meda's Aldara, indicated in the topical treatment of clinically typical, non-hyperkeratotic, non-hypertrophic, visible, or palpable actinic keratosis (AK) of the full face or balding scalp in immunocompetent adults when other topical treatment options are contraindicated or less appropriate.
In addition to the above, the committee adopted a negative opinion on Pfizer's Elelyso, not recommending it for marketing authorisation.
Outlook and Implications
At its June meeting, the CHMP issued a positive opinion on four drugs—Cuprymina, Revestive, Enurev Breezhaler/Seebri Breezhaler/Tovanor Breezhaler, and Zinforo—as well as recommendations for the extension of therapeutic indications of four drugs: Afinitor, Cayston, Enbrel, and Humira.
The above issued recommendations will be sent to the European Commission for authorisation; if confirmed, the drugs will be granted marketing authorisation in the EU-27 countries plus Iceland and Norway.
The committee's decision not to recommend Elelyso for marketing authorisation in the EU is because another drug, Vpriv (velaglucerase alpha; Shire, UK), indicated for the same condition, has already been approved and thus qualifies for 10-year marketing exclusivity in the EU for this orphan indication. Vpriv received authorisation in August 2010 (see United Kingdom: 27 August 2010: Shire Gains Final EU Approval for Vpriv). Under EU orphan drug-development incentives, "another drug cannot be authorised for the same condition if it is similar to the medicine already authorised". The decision is a blow to Pfizer as well as to its Elelyso development partner Protalix. Elelyso has recently achieved US FDA approval in the US, despite the six-year marketing exclusivity on orphan drugs (see United States: 2 May 2012: Pfizer Gains FDA Approval on Elelyso for Gaucher, Lipitor and Celebrex Hang over Q1 Results). With the setback in marketing authorisation in the EU, the collaboration would have to focus on other regions.
Furthermore, the CHMP can issue scientific opinion, as part of its co-operation with the World Health Organisation, for medicines to be used outside the EU. The current recommendation for vaccine Hexaxim falls under this category. The recommendations by the committee for drugs for use outside the EU are not sent to the European Commission.