Two small European biotech companies, Pharming and Archimedes, had their lead products, Ruconest and PecFent respectively, recommended for approval by the EU’s Committee for Medicinal Products for Human Use at its monthly meeting on June 21-24.
The committee also gave positive opinions on Shire’s Vpriv for Gaucher’s disease and Merck & Co’s atrial fibrillation therapy Brinavess and its bipolar disorder product, Sycrest.
Ruconest for HAE
Pharming’s Ruconest (conestat alfa), a human recombinant protein extracted from the milk of transgenic rabbits, was recommended for the acute treatment of angioedema attacks in patients with hereditary angioedema (HAE).
The orphan product, a recombinant human C1 inhibitor formerly known as Rhucin, will be marketed by two partners – Dr Esteve in Spain, Portugal, Greece and Andorra, and Swedish Orphan Biovitrum in all other EU countries and in Iceland, Norway and Liechtenstein.
Pharming estimates the current value of the European HAE market at €110 million ($136 million). Competing products marketed in Europe are CSL Behring’s plasma-derived C1 inhibitor concentrate, Berinert P and Shire’s bradykinin antagonist, Firazyr (icatibant).
Ruconest was previously rejected by the CHMP in December 2007, when the committee said there was insufficient data to support its approval. The company refiled the product with additional data from a recently completed North American Phase III trial.
PecFent For Cancer Pain
Archimedes Pharma submitted a “hybrid generic” marketing authorisation for its nasal spray, PecFent (fentanyl), because it consists of a generic product, fentanyl, in a new pharmaceutical formulation, a nasal spray. Other companies market in the EU different formulations of fentanyl – Actiq lozenges and Effentora buccal tablets.
PecFent is the first product to use Archimedes’ proprietary PecSys technology, which improves the retention of fentanyl on the nasal mucosa, according to the company. The CHMP has given a positive opinion to its use in the treatment of breakthrough cancer pain. A US NDA has been filed and is awaiting approval, the company said.
Archimedes already markets a range of products for other companies in the UK, France, Germany and Ireland, including Gliadel, Zomorph and Oramorph, and says it will launch its own commercial organization in the US when PecFent is approved there.
Accelerated Vpriv Assessment
Shire’s Vpriv (velaglucerase alfa) was approved for use in the treatment of Gaucher’s disease via an accelerated assessment. This was due to the “major public health interest” in doing so, the CHMP said, because of the ongoing shortages of Genzyme’s marketed Gaucher’s disease product, Cerezyme (imiglucerase), due to manufacturing problems.
Vpriv was first approved in the US in February, and Shire said that with the earlier than expected adoption of Vpriv worldwide, is was starting a program with physicians and patients to monitor and manage requests from new patients carefully, in order to ensure the long-term uninterrupted treatment with Vpriv.
Vernakalant For AF
Merck & Co has received a positive CHMP recommendation for the intravenous atrial fibrillation product, Brinavess (vernakalant), which it has licensed from the Canadian biotech, Cardiome Pharma. It is the first of a number of new cardiovascular products that the company is developing.
Merck has exclusive rights to intravenous vernakalant outside of the US, Canada and Mexico, and exclusive global rights to the oral formulation of vernakalant, following a $800 million deal signed last year.
Intravenous vernakalant was recommended for the rapid conversion of recent-onset atrial fibrillation to sinus rhythm in adults: for non-surgery patients with atrial fibrillation of 7 days or less, and for post-cardiac surgery patients with atrial fibrillation of 3 days or less.
The product is expected to compete with generic anti-arrhthymics like amiodarone and newer agents like Sanofi-Aventis’s Multaq (dronedarone). In one clinical trial, the AVRO study, vernakalant was studied in 116 patients with atrial fibrillation (which had been present for 3 hours to 48 hours) versus 116 patients treated with amiodarone. Vernakalant converted 51.7 percent of patients to sinus rhythm at 90 minutes whereas amiodarone converted 5.2 percent of patients.
A second Merck product, the antipsychotic Sycrest (asenapine), was recommended for approval for the treatment of moderate to severe manic episodes associated with bipolar I disorder in adults.
Asenapine has been recommended for a narrower indication in the EU than that approved last year in the US, where it was approved both for the first-line treatment of schizophrenia and acute treatment of manic or mixed episodes associated with bipolar I disorder with or without psychotic features in adults.
Zeftera Rejection Confirmed
The negative opinon on Janssen-Cilag’s intravenous antibiotic, Zeftera (ceftobiprole, licensed from Basilea), issued by the CHMP in February, was confirmed following an appeal by the company.
The February opinion had reversed a previous positive opinion issued in November 2008. But the CHMP later received information from the US FDA indicating that some study sites had not performed clinical studies in compliance with good clinical practice. The committee looked at further analyses of the data from the clinical studies and inspections, but due to continuing concerns over the reliability of the study results, the CHMP refusal was confirmed.
- John Davis (j.davis@elsevier.com)
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