Celgene International Sàrl, a wholly owned subsidiary of Celgene Corporation (NASDAQ:CELG) announced that that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for an expanded indication of VIDAZA® (azacitidine for injection) for the treatment of adult patients aged 65 years or older with acute myeloid leukaemia (AML) who are not eligible for haematopoietic stem cell transplantation (HSCT). The expanded indication now covers patients who have > 30% myeloblasts according to the WHO classification; previously, the indication covered AML patients with < 30% blasts.

Myeloblasts are white cells in the bone marrow; in AML, their functioning is disrupted and results in numerous non-functioning white cells, which can potentially interfere with the body’s ability to control infections and can lead to anaemia and haemorrhages.

For many patients, AML is typically associated with a poor prognosis and deteriorating quality of life, particularly for those patients who cannot tolerate curative therapies like stem cell transplantation. In Europe, more than 14,000 people suffer from AML, and most of these patients will die within less than 1 year. As an acute leukaemia, AML progresses rapidly and is typically fatal within months if stem cell transplant is not an option. Specific to elderly patients, overall survival with AML has not improved in more than 40 years1, and there is a clear need for treatments that can support this patient population.

“While progress has been made in treating younger, fitter AML patients who can undergo intensive and potentially curative therapies such as stem cell transplant, there is still a clear need for treatments for elderly and more frail patients,” said Hervé Dombret, M.D., Chief, Blood Disease Department (Leukaemia Unit), University Hospital Saint-Louis, AP-HP, Paris, France. “Azacitidine has demonstrated a median overall survival of 10.4 months, and these results suggest that, if approved, azacitidine could provide a valuable treatment option for patients who have limited options today.”

Adds Tuomo Pätsi, President of Celgene in Europe, Middle East and Africa (EMEA): “Celgene is committed to bringing innovative medicines to patients with haematological diseases including AML. With the positive CHMP opinion for VIDAZA in AML, Celgene has an opportunity to advance the treatment options available to patients with AML. And, we will continue to focus on meeting the unmet needs of patients with myeloid disease, as we have several partnerships and development programmes that will build on what we are learning about treating these diseases.”

The CHMP decision was based on data from AML-001, a global, multi-centre, randomized, open-label pivotal study of patients at least 65 years old with newly diagnosed or secondary AML with > 30% bone marrow blasts. VIDAZA plus best supportive care (n=241) was compared with conventional care regimens (n=247). Median overall survival (OS), the primary endpoint of the study, was 10.4 months (95% CI 8.0-12.7 months) for patients receiving azacitidine compared with 6.5 months (95% CI: 5.0-8.6) for patients receiving conventional treatment regimens (HR=0.85 [95% CI 0.69, 1.03], stratified log-rank p=0.1009). One-year survival rates with azacitidine and conventional treatment regimens were 46.5% and 34.2%, respectively (difference 12.3% [95% CI: 3.5% – 21%]).

In the study, grade 3-4 anaemia, neutropenia, febrile neutropenia, and thrombocytopenia rates, respectively, were 16%, 26%, 28%, and 24% with azacitidine; 5%, 5%, 28%, 5% with best supportive care; 23%, 25%, 30%, 28% with low-dose Ara-Cytarabine; and 14%, 33%, 31%, 21% with intensive chemotherapy.

In addition to recommending the marketing authorisation for the new indication to the European Commission, the CHMP also noted that this new therapeutic indication brings significant clinical benefit in comparison with existing therapies; if the European Commission adopts the CHMP decision in full, VIDAZA will receive extended market protection in all its indications for an additional year throughout the European Economic Area.

The CHMP reviews applications for all 28 member states in the European Union (EU), as well as Norway, Liechtenstein and Iceland. The European Commission, which generally follows the recommendation of the CHMP, is expected to make its final decision within two months. If approval is granted, detailed conditions for the use of this product will be described in the Summary of Product Characteristics (SmPC), which will be published in the revised European Public Assessment Report (EPAR).

The anticipated European Commission decision would add to the portfolio of indications VIDAZA is authorised for across high-risk myeloid diseases, including myelodysplastic syndromes (MDS) and AML. VIDAZA has been approved in the EU since 2008 for the treatment of adult patients ineligible for transplantation diagnosed with intermediate 2 and high-risk MDS; chronic myelomonocytic leukaemia (CMML) with 10-29 % marrow blasts without myeloproliferative disorder; or acute myeloid leukaemia (AML) with 20-30 % blasts and multi-lineage dysplasia.

In the United States, VIDAZA is not indicated for treatment of patients with AML. VIDAZA is indicated for treatment of patients with the following French-American-British (FAB) myelodysplastic syndrome subtypes: refractory anaemia (RA) or refractory anaemia with ringed sideroblasts (RARS) (if accompanied by neutropenia or thrombocytopenia or requiring transfusions), refractory anaemia with excess blasts (RAEB), refractory anaemia with excess blasts in transformation (RAEB-T), and chronic myelomonocytic leukemia (CMMoL). (Original Source)

Shares of Celgene Corporation closed yesterday at $113.88. CELG has a 1-year high of $140.72 and a 1-year low of $83.16. The stock’s 50-day moving average is $122.90 and its 200-day moving average is $119.72.

On the ratings front, Celgene has been the subject of a number of recent research reports. In a report issued on September 1, Raymond James analyst Christopher Raymond initiated coverage with a Buy rating on CELG. Separately, on July 27, Morgan Stanley’s Matthew Harrison maintained a Hold rating on the stock and has a price target of $134.

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Christopher Raymond and Matthew Harrison have a total average return of 46.3% and 8.0% respectively. Raymond has a success rate of 78.6% and is ranked #16 out of 3764 analysts, while Harrison has a success rate of 51.9% and is ranked #1042.

The street is mostly Bullish on CELG stock. Out of 15 analysts who cover the stock, 12 suggest a Buy rating and 3 recommend to Hold the stock. The 12-month average price target assigned to the stock is $157.27, which implies an upside of 38.1% from current levels.

Celgene Corp is a biopharmaceutical company. It is engaged in the discovery, development and commercialization of therapies designed to treat cancer and immune-inflammatory related diseases.