Novartis’ biological medicine Ilaris (canakinumab) has become the first drug approved in the EU for patients as young as four years old with cryopyrin-associated periodic syndrome (CAPS).

The approval of Ilaris as an orphan drug is based on trial evidence that it produced rapid and sustained remission of CAPS symptoms in up to 97% of patients.

CAPS is a rare lifelong auto-inflammatory disease with debilitating symptoms and few treatment options. Monoclonal antibody Ilaris selectively targets and blocks interleukin-1 beta (IL-1ß), the trigger for inflammation and tissue damage in CAPS patients.

Ilaris is the only medicine approved in the EU for CAPS patients as young as four years old, and for patients with the most debilitating form of CAPS, neonatal-onset multisystem inflammatory disease (NOMID).

“We are excited by the latest approval because Ilaris represents a significant therapeutic advance for patients with this debilitating and sometimes fatal disease,” said Joe Jimenez, CEO of the Novartis Pharmaceuticals Division. “Ilaris is the outcome of our pathways-driven search for innovative medicines that are tailored to the needs of patients.

“Initially we studied Ilaris in a very rare disease with a well-understood genetic profile, and now that its efficacy has been proven, we are able to move ahead rapidly with development in other diseases characterised by the same inflammatory process.”

The regulatory submission was supported by data showing that Ilaris produced rapid and sustained remission of symptoms in up to 97% of CAPS patients, with most of them responding within hours of the first injection.

“In CAPS studies, symptoms improved within 24 hours after patients received a single dose of Ilaris. The disease was barely detectable in the blood after two weeks and the remission of symptoms was sustained for six months,” said Helen J. Lachmann, MD of the UK National Amyloidosis Centre at the Royal Free and University College Medical School in London. “By effectively turning off the disease activity, Ilaris has the potential to transform patients’ lives by offering long-term control of their disease.”

The accelerated EU decision follows approvals in the US and Switzerland, where Ilaris was granted priority review in view of the significant unmet medical need.

Studies with Ilaris are ongoing in other diseases in which IL-1ß plays an important role, such as chronic obstructive pulmonary disease (COPD), type 2 diabetes, systemic juvenile idiopathic arthritis (SJIA), and gout.

New data show that patients have higher overall treatment satisfaction with one daily injection of Victoza (liraglutide, 1.8mg dose) than they do with two daily injections of exenatide (10μg dose).

The data from the LEAD6 trial was recently presented at the 20th World Diabetes Congress (International Diabetes Federation).

Specifically, among the 379 patients who completed the Diabetes Treatment Satisfaction Questionnaires (DTSQ) during the trial, those taking once-daily Victoza perceived less hypoglycaemia (abnormally low blood sugar levels) or hyperglycaemia (abnormally high blood sugar levels) compared to those on twice-daily exenatide.

“The study shows Victoza is associated with higher patient satisfaction compared to exenatide,” said Professor Bain, Chair in Medicine (Diabetes) at Swansea University and Diabetes Lead Clinician for the ABM University Health Board.

“It is important to consider patient-reported outcomes data, because if a patient is satisfied with his or her treatment they are more likely to comply with the treatment over the long term, which is necessary in type 2 diabetes management,” he added.

Treatment satisfaction was also evaluated in patients who were either switched from twice-daily exenatide to once-daily Victoza or continued on once-daily Victoza for another 14 weeks. In these cases, switched patients had improved satisfaction compared to those who continued on Victoza from weeks 26-40.

NICE has recommended the use of antidepressants to treat patients with moderate or severe depression, or for those with mild symptoms when other interventions have not been successful.

However, the Institute has urged against the routine use of antidepressants to treat patients with only mild symptoms.

These recommendations form part of updated guidance, produced for NICE by the National Collaborating Centre for Mental Health, on the identification, treatment and management of depression in adults in both primary and secondary care.

The guidance states that drug treatment should be considered for patients with a past history of moderate or severe depression, for those with sub-threshold depressive symptoms that have been present for at least 2 years, or for patients with mild depression that persists after other interventions.

For those with mild symptoms, the guidelines recommend initial treatment with either individual guided self-help based on the principles of cognitive behavioural therapy (CBT), computerised cognitive behavioural therapy (CCBT) or a structured group physical activity programme.

For the treatment of moderate or severe depression, NICE encourages a combination of antidepressant medication and a high-intensity psychological intervention such as CBT.

The guidance also adds that patients who have benefited from antidepressant treatment should be supported to continue treatment for at least six months after symptoms have improved, to reduce the risk of relapse.

Professor Ian Anderson, Professor of Psychiatry, University of Manchester and Chair of the Guidance Development Group (GDG), said: “The treatment of depression can be difficult. Everyone who has depression has their own story and their treatment needs to be individually tailored for them.

“These guidelines offer choice and will support doctors and those with depression to find the best treatment for them. Good communication between doctors and their patients is essential and the decision as to which treatments to use should be a decision made in partnership.”

NICE has published final guidance recommending the use of Lilly’s Effient (prasugrel) in combination with aspirin as an option for preventing blood clots in the arteries of people with acute coronary syndromes.

Acute coronary syndromes are a group of heart problems which occur due to narrowed coronary arteries. These heart problems cause chest pain and specific types of heart attack.

Prasugrel is a drug that can reduce or prevent the formation of blood clots in arteries by reducing the clumping together of platelets in the blood.

The guidance recommends prasugrel in the above indication, but only when immediate primary percutaneous coronary intervention for ST-segment-elevation myocardial infarction (a specific type of heart attack) is necessary or a blood clot blocking a stent has occurred during treatment with clopidogrel.

It is also approved for patients with acute coronary syndromes who have diabetes mellitus.

Dr Carole Longson, Director at NICE, said: “There are a range of heart problems classed as acute coronary syndromes – these conditions have a huge impact on the individuals affected. This new guidance recommending prasugrel in combination with aspirin will offer an additional treatment option for these specific individuals at risk of blood clots.”

Shire’s phosphate binder Fosrenol (lanthanum carbonate) has gained approval in the EU for an extension of indication through the European Mutual Recognition Procedure.

This approval paves the way for Fosrenol’s use to control hyperphosphataemia in chronic kidney disease (CKD) patients who are not on dialysis, with a serum phosphorus level greater than or equal to 1.78mmol/L (5.5mg/dL).

The extension was sanctioned by the Swedish Medicines Products Agency as reference member state.

“Failure to control phosphate in the earlier stages of chronic kidney disease carries well-documented risks, and is associated with reduced bone health and poor cardiovascular outcomes,” said Dr Alastair Hutchison, Manchester Royal Infirmary.

“The extension to the existing indication for Fosrenol provides nephrologists in the EU with an important additional option to help tackle the challenge of uncontrolled phosphate at an earlier stage in the progression of kidney disease, before the need for dialysis treatment.”

“This is an important development in helping CKD patients better manage their elevated phosphate and we are pleased that Fosrenol is now approved as a treatment option for these patients in the EU,” added Gian Piero Reverberi, Senior Vice President, International Specialty Pharmaceuticals, Shire. “We are firmly committed to serving the needs of renal patients and ensuring that Fosrenol is available to the prescribers and patients who can benefit from it.”

Dr Carole Longson

GSK’s oral cancer treatment Hycamtin (topotecan) has received its second NICE recommendation this month.

Final NICE guidance has been published supporting the drug in combination with cisplatin as a treatment option for women with recurrent or stage IVB cervical cancer who have not previously received cisplatin.

This is in addition to Hycamtin’s recommendation for relapsed small-cell lung cancer earlier this month.

Topotecan prevents DNA replication in cancer cells by inhibiting the enzyme topoisomerase I, which helps to separate and repair the DNA in cells when they divide.

Dr Carole Longson, Health Technology Evaluation Centre Director, said: “The independent Appraisal Committee considered all available evidence on the effectiveness of topotecan as a treatment for women with cervical cancer. They also heard evidence from patients who highlighted the importance of having a number of treatment options because one may be more suitable than others for the individual patient.

“The committee concluded that – for women with recurrent cervical cancer and those with stage IVB cervical cancer, where cancerous cells have spread to distant organs, such as the lungs, and who have not previously received cisplatin – the cost of topotecan in relation to how well it works is an effective use of NHS resources.”

NICE has added that women who have previously received cisplatin and are currently being treated with topotecan in combination with cisplatin should have the option to continue their therapy until it is considered appropriate to stop.

Norgine has appointed Lance Richard as Global Market Access Manager, within its Global Marketing department.

His primary responsibilities are to support pricing, reimbursement and market access for Norgine brands and to demonstrate value across the brand lifecycle.

Lance started his career as a clinical pharmacist in South Africa, before moving into the field of health economics and outcomes research.

In his capacity as a health economist, he has worked both in industry and consultancy, having served as Senior Health Economist, Global Health Outcomes Research at UCB Celltech and, more recently, as Director of Market Access at GfK Healthcare.

He studied clinical pharmacy at the University of the Witwatersrand in Johannesburg, South Africa, and has a degree in health economics from the University of York, UK.

The CHMP has issued a positive opinion for Angiox (bivalirudin) for use in heart attack patients undergoing emergency heart procedures.

The recommendation could extend the use of Angiox (bivalirudin) to include patients with heart attacks (so-called ST segment elevation myocardial infarction) undergoing an emergency heart procedure called primary percutaneous coronary intervention (PCI).

The basis of the approval is the HORIZONS-AMI study, the first drug trial to demonstrate a reduction in deaths from heart attacks in patients undergoing emergency PCI. The trial showed that patients treated with Angiox compared with today’s leading treatment – heparin plus a platelet glycoprotein IIb/IIIa inhibitor (GPI) – were more likely to survive and had less frequent severe bleeds.

Professeur Ph. Gabriel Steg, from Centre Hôpitalier Bichat in Paris, France, commented: “The sustained reduction in all-cause and cardiovascular mortality achieved by bivalirudin in the context of primary PCI, compared to the standard of care, provides a compelling case for switching to bivalirudin as the preferred anticoagulant for primary PCI.”

The HORIZONS-AMI trial compared Angiox to heparin plus a GPI in 3,602 patients presenting with the most severe form of heart attack, ST segment elevation myocardial infarction (STEMI), undergoing an emergency (primary) PCI. Results at 30 days showed that Angiox significantly improved overall mortality, including a reduction in the incidence of heart-related deaths by 38% (1.8% vs. 2.9%).

Angiox also reduced the incidence of major bleeding by 42%, reduced the incidence of adverse clinical events by 26%, and demonstrated comparable rates of major adverse cardiac events.

Long-term results of the data have recently been published, including one-year data in The Lancet and two-year data presented at the 2009 Transcatheter Cardiovascular Therapeutics (TCT) conference. These data show a significant 38% reduction in cardiac mortality at 30 days, which was maintained at 43% (at one year) and 41% (at two years).

“The HORIZONS-AMI trial confirms the role of Angiox as an alternative anticoagulant strategy to treat patients with acute myocardial infarction undergoing primary PCI. The safety benefit observed with bivalirudin against unfractionated heparin + GPIIb/IIIa inhibitors is a real contribution to interventional cardiology in the contemporary era of evidence-based medicine,” said Professor Gilles Montalescot of the Pitié-Salpêtrière Hôpital, France.

Actelion UK has appointed Robin Bhattacherjee to the role of General Manager for the UK and Ireland, based at the Company’s UK office in London.

Prior to this role, Robin was Executive Director at CV Therapeutics (CVT) Europe, where he was responsible for establishing the European office for CV Therapeutics.

At CVT he was also responsible for spearheading the strategic development and launch of a range of specialist cardiovascular products and identifying and establishing CVT European marketing partnerships before CVT was acquired by Gilead Sciences in 2009.

With a pharmaceutical career history spanning over two decades, Robin brings a wealth of knowledge and experience to Actelion UK from several managerial roles, which include Head of Cardiovascular Marketing at Sanofi-Synthélabo UK and Marketing Director within Bayer’s Global Strategic Marketing Group.

Robin commented: “This role attracted me from the very outset and I am privileged to have the opportunity to lead the UK team of such an innovative global pharmaceutical company. I look forward to supporting Actelion’s continued commitment to these important therapy areas, to the specialist physicians, healthcare professionals and patient group communities.”

Actelion Pharmaceuticals is committed to researching and developing treatments for rare orphan diseases in areas of high unmet clinical need, such as pulmonary arterial hypertension, Gaucher’s and Niemann-Pick type C. The company’s first drug, Tracleer, has been approved as a therapy for pulmonary arterial hypertension in patients with functional class III in the UK.

The European Union has approved Merck’s proposed takeover of Schering-Plough, which would create the second biggest global producer of prescription medicines.

The EU’s antitrust authorities said in a statement that the “transaction would not significantly impede effective competition” in Europe.

The $41.1 billion acquisition of Schering-Plough will make Merck the second largest prescription drug company, with about $42.4 billion in annual sales.

Integration planning teams from the two companies are working together to ensure that the combined company will be well equipped to begin its first day of business as soon as the merger is completed.

“Approval from the European Commission marks a key milestone for the completion of our transaction with Schering-Plough,” said Merck President and CEO Richard T. Clark.

“We’re making good progress towards our closing and look forward to creating a strong global leader that will make a substantial difference to patients and healthcare around the world.”

The two companies hope to close the deal in the fourth quarter after shareholders approved it in August. It still needs approval from the US Federal Trade Commission.

In its checks for overlaps in Europe, specifically in the areas of asthma and allergic rhinitis, the EU Commission found the products were not close competitors and that the tie-up would not prevent other companies from competing.