Merck KGaA to appeal negative European decision on multiple sclerosis drug cladribine

Merck KGaA is to request a re-examination of the negative opinion issued last month by European regulators regarding its oral drug cladribine for the treatment of relapsing-remitting multiple sclerosis (MS), the company reported Monday.
In September, the European Medicines Agency's Committee for Medicinal Products for Human Use indicated that based on data included in the company's regulatory filing, the benefits of cladribine do not outweigh its risks. At the time, Elmar Schnee, head of Merck's drug unit, noted the agency raised concerns about cases of cancer seen in clinical trials of the drug, as well as cases of lymphopenia.
A decision on the drug, which has been approved in Russia and Australia, in the US is expected in the fourth quarter of the year, after the company's initial regulatory filing was rejected by the FDA as incomplete.
Last month, Novartis' Gilenya (fingolimod) became the first oral drug to be approved in the US as a first-line treatment for relapsing forms of MS, while a decision on the product in Europe is anticipated during the next six months.

Reference Articles
Merck KGaA to appeal European panel view on cladribine - (FOX Business)
Merck KGaA to ask EU agency to reconsider MS pill - (Forexpros)
Merck KGaA notified EMA of its intention to appeal CHMP opinion on cladribine tablets in MS - (Merck KGaA)

**Published by "First Word"

Novartis reports Afinitor neuroendocrine tumour data, filings expected this year

Novartis reported Monday data at the European Society for Medical Oncology congress from a late-stage trial in which Afinitor (everolimus) extended median progression-free survival (PFS) in patients with advanced neuroendocrine tumours (NET) when compared to placebo. The Swiss company noted that results from the RADIANT-2 study, along with those from another Phase III trial, will form the basis of worldwide regulatory filings this year for the mTOR inhibitor.
The RADIANT-2 study enrolled 429 patients with advanced NET who were randomised to receive Sandostatin LAR (octreotide acetate) in combination with either once-daily Afinitor or placebo. Although the trial failed to meet its primary endpoint of PFS, Novartis said data showed patients treated with Afinitor had a "clinically meaningful" increase in median PFS of 16.4 months, compared with 11.3 months for those who took placebo.
Lead researcher Marianne Pavel commented that the study was designed to test "for an improvement of at least 4.5 months compared to [Sandostatin] LAR and were pleased with the resulting 5.1 month difference." The drugmaker also indicated that after adjusting for "imbalances in the treatment arms," Afinitor was shown to significantly reduce the risk of disease progression by 40 percent compared to placebo.
The company also presented data at ESMO from the < href="http://www.firstwordplus.com/Fws.do?articleid=CA1B497D623442D4A570A45D338B85E4">previously reported RADIANT-3 trial, which demonstrated that Afinitor extended the median time without tumour growth from 4.6 to 11 months when compared with placebo in patients with advanced pancreatic NET.
Afinitor was approved last year in both the US and Europe as a treatment for patients with advanced renal cell carcinoma. Analysts expect that yearly sales of the drug could reach $1 billion by 2014.

Reference Articles
Novartis's Afinitor fails to slow rare form of pancreatic cancer in trial - (Bloomberg)
Phase III Novartis data show potential benefit of Afinitor plus Sandostatin LAR in patients with advanced neuroendocrine tumors - (Novartis)
Novartis to file Afinitor after Phase III study data (free preview) - (The Wall Street Journal)
Novartis drug helps rare tumors, filing this year - (Yahoo!News)

**Published in "First Word"

Report: CEO says Bristol-Myers Squibb to stick with "big pharma legacy"

According to Bristol-Myers Squibb CEO Lamberto Andreotti, the company will continue to pursue its strategy of developing innovative prescription drugs rather than diversifying as other pharmaceutical companies have done, the Financial Times reported Monday. Andreotti, who replaced James Cornelius in May, said he plans to "not give up the big pharma legacy, and combine it with what’s good in biotech," adding that he would pursue "selective integration," as well as partnerships to share risk, cost and expertise. "We are not going to do everything ourselves," he noted.

Reference Articles
Bristol-Myers Squibb continues tradition - (Financial Times)

**Published in "First Word"

Johnson & Johnson's abiraterone acetate increases survival in prostate cancer trial

According to preliminary data from a late-stage trial presented at the European Society for Medical Oncology congress Monday, Johnson & Johnson's experimental drug abiraterone acetate, when combined with prednisone, increased median survival time of patients with advanced prostate cancer to 14.8 months, compared to 10.9 months for those who took the steroid alone. Last month, the drugmaker said that all patients participating in the study would be offered the drug after based on an interim analysis.

Reference Articles
Trial confirms prostate drug promise - (BBC News)
J&J drug for prostate cancer extends life in study - (Bloomberg)
New drug offers hope in advanced prostate cancer - (The Post Chronicle)
Prostate cancer drug a 'significant move forward' - (The Telegraph)
J&J prostate cancer drug prolongs survival - (TheStreet.com)

**Published in "First Word"

Según un estudio europeo sólo una de cada cinco madres ponen reparo a las vacunas

El proyecto europeo 'VACSATC', en el que participan investigadores de la Universidad de Alicante (UA), ha revelado que el 20,9 por ciento de las madres en España manifiesta alguna preocupación sobre la seguridad de las vacunas y las madres españolas son, además, más reacias a las vacunas de pago, según ha informado la institución académica.
Esta son algunas de las conclusiones de este estudio, publicado en la revista 'Vaccine', y que ha estudiado las opiniones de padres y madres sobre la vacunación de sus hijos en España, Inglaterra, Polonia, Noruega y Suecia entre mayo de 2008 y diciembre de 2009. Así, de las 6.611 encuestas realizadas, 913 pertenecían a madres españolas de niños y niñas de entre 0 y 3 años realizadas por medio de entrevistas cara a cara.
El investigador de la UA en el proyecto europeo 'VACSACTC' José Tuells ha señalado que "las madres se muestran más preocupadas en España por la vacunación de sus hijos desde que se produjo el incidente con las vacunas contra el virus del papiloma humano (VPH)".
En este sentido, según el trabajo, el 20,9 por ciento de las madres encuestadas en España ha tenido alguna vez preocupación sobre la seguridad de las vacunas, frente al 4,1 por ciento en Noruega.
En el estudio, el investigador ha indicado que se compararon las fuentes que utilizan los padres para obtener información sobre las vacunas, a quienes les solicitaron que valoraran cuáles eran las más fiables y su opinión sobre el papel de la vacunación, la seguridad de las vacunas en sus países y qué enfermedades consideran más peligrosas.
SERVICIOS DE VACUNACIÓN
Los resultados obtenidos apuntan también que España es el país donde las madres manifiestan menos dudas a la hora de vacunar a sus hijos, ya que el 90,8 por ciento muestra su satisfacción con los servicios de vacunación.
Respecto a si van a continuar vacunando a sus hijos en un futuro, todos los países muestran una alta aceptación, superior al 90 por ciento, a excepción de Inglaterra, cuya cifra desciende al 80,9 por ciento.
"Este hecho se debe a que la población inglesa arrastra la polémica de la vacuna de la triple vírica -sarampión, rubéola, paperas- desde hace más de 10 años y hay un rechazo a esta vacuna", ha explicado Tuells.
Por otro lado, el estudio ha revelado que todos los países han confirmado que son los profesionales de la salud los más adecuados como fuente de información sobre vacunas.
"El gobierno y los institutos nacionales de salud pública también se señalaron como fuentes de información importantes y de confianza en Noruega, Suecia e Inglaterra", ha apuntado el investigador, quien ha explicado que en España y Polonia, los medios de comunicación, la familia y los amigos son la segunda fuente de confianza más usada.

UPDATE 2-FDA declines approval of Jazz drug for fibromyalgia

Jazz Pharmaceuticals Inc (JAZZ.O) said the U.S. health regulator did not approve its experimental drug to treat the pain disorder fibromyalgia and suggested the need for additional clinical studies, sending its shares down 6 percent in pre-market trade.
"This news does not come as a surprise to us... I think it will be a long way for fibromyalgia, with several years of delay," Capstone Investments analyst Difei Yang said.
The U.S. Food and Drug Administration's (FDA) complete response letter said the drug, JZP-6, could not be approved in its present form and "discusses a number of topics, including the need for additional clinical studies," Jazz Pharma said in a statement.
In August, a U.S. advisory panel rejected the drug for fibromyalgia -- a poorly understood disorder that causes pain and fatigue -- amid concerns the product could be misused as a "date-rape" drug. [ID:nN20135341]
"Based on the committee members' inputs at the time of the panel meeting, I think there will be an additional clinical trial, we don't know the scope of the clinical trial but another trial means the drug will be delayed by at least two years," the analyst added.
The complete response letter sent to the company also addresses issues like the appropriate patient population, methods for ensuring safe use, the proposed risk evaluation and mitigation strategy, concentration and trade name for the product.
The analyst, who did not change her rating on the stock, said another trial could cost the company in millions and there could be a possibility the company suspends the program altogether.
"However, Jazz should not make any hasty decision and I think they will take time to decide," Difei added.
The company said it has requested a meeting with the FDA to discuss and clarify the contents of the letter.
Jazz Pharma's shares fell 27 percent in the first trading session after the panel rejected the drug on Aug. 20. They have regained most of their losses since then to close at $10.11 Friday on Nasdaq. The stock was down 6 percent at $9.46 in pre-market trade on Monday.

**Published in "Reuters"

FDA issues complete response letter for Alexza Pharmaceuticals anti-psychotic drug

Alexza Pharmaceuticals on Monday announced that that it received a complete response letter from the FDA regarding its application for AZ-004 for the rapid treatment of agitation in schizophrenia or bipolar disorder patients. In the letter, the agency stated that the application is not ready for approval in its present form due to safety concerns.

Reference Articles
Alexza Pharmaceuticals receives Complete Response Letter for AZ-004 (Staccato loxapine) NDA - (Alexza)
Alexza say FDA declines inhaled agitation drug - (CNBC)
Alexza shares sink as the FDA denies its antipsychotic drug - (Daily Finance)
Alexza plunges on FDA setback; drug indexes flat - (MarketWatch)
Alexza to meet with FDA to address concerns on bipolar drug - (The Wall Street Journal)
Alexza Pharm: FDA doesn't approve AZ-004 - (TheStreet.com)

**Published in "First Word"