Medindia LOGIN REGISTER
Medindia
Advertisement

Nplate(TM) (Romiplostim) Receives Positive Opinion for Marketing Authorisation in the European Union

Saturday, November 22, 2008 General News
Advertisement
THOUSAND OAKS, Calif., Nov. 21 Amgen(Nasdaq: AMGN) today announced that the European Committee for MedicinalProducts for Human Use (CHMP) has issued a positive opinion recommendingmarketing authorisation for Nplate(TM) (romiplostim) in the European Union(EU). The CHMP recommends Nplate for adult chronic immune (idiopathic)thrombocytopenia purpura (ITP) splenectomised patients who are refractory toother treatments (e.g. corticosteroids, immunoglobulins). Nplate may beconsidered as second line treatment for adult non-splenectomised patientswhere surgery is contra-indicated.
Advertisement

"Nplate will address an unmet medical need for thousands of patients inthe European Union as it is a unique treatment option that increases plateletproduction and avoids immune suppression in adult chronic ITP patients," saidWillard Dere, M.D., senior vice president and international chief medicalofficer at Amgen.
Advertisement

The novel peptibody technology upon which romiplostim is based representsa promising new approach for treating adult patients with chronic ITP, anautoimmune disorder affecting an estimated 50,000 people in the EU, which canlead to serious bleeding events that can be potentially life threatening.

Nplate, a thrombopoietin (TPO) mimetic, is a novel engineered therapeuticfusion protein with attributes of both peptides and antibodies, but isdistinct from each. Nplate works similarly to TPO, a natural protein in thebody. Nplate stimulates the TPO receptor, which is necessary for growth andmaturation of bone marrow cells that produce platelets.

The CHMP positive opinion is based on data from two separateplacebo-controlled Phase 3 studies, demonstrating that platelet counts wereraised and sustained in 83 percent of patients for both splenectomised andnon-splenectomised groups when treated with Nplate. Additionally, patientstreated with Nplate were able to reduce or discontinue concomitant ITP andemergency medications which are often not well tolerated or whose effects aretransient (i.e. corticosteroids, IVIG, Win-Rho Anti-D therapy).

Upon completion of the Phase 3 studies almost 90 percent of patientselected to subsequently enroll into the romiplostim long term extension studywhich demonstrated that, after three years, Nplate continued to effectivelyincrease and sustain platelet counts. In this open label long term extensionstudy some patients were treated for over 156 weeks and in the interimanalysis the median treatment duration in this study is 65 weeks.

About Adult ITP

In patients with ITP, platelets -- or blood cells needed to preventbleeding -- are destroyed by the patient's own immune system. Low plateletcounts leave adult ITP patients open to sudden serious bleeding events, makingit impossible to arrest blood flow. The risk for serious bleeding eventsincreases when platelet counts drop to less than 30,000 platelets permicroliter; normal counts range from 150,000 to 400,000 platelets permicroliter. ITP has historically been considered a disease of plateletdestruction although recent data suggest that the body's natural plateletproduction processes in ITP are unable to compensate for low levels ofplatelets in the blood. Increasing the rate of platelet production mayaddress low platelet levels associated with ITP.

Currently available treatments (i.e., corticosteroids, immunoglobulins)have limited application due to poor tolerability or transient effects.Surgical therapy (removal of the spleen) is also available to adult patientswith chronic ITP, but does not work in all cases. Currently, there are140,000 treated chronic ITP patients in Europe and the U.S. ITP affects abouttwice as many adult women as men.

About Nplate

Nplate was granted approval for ITP by the regulatory bodies in Australiain July and the United States (U.S.) in August 2008. In addition to theEuropean Union (EU), Amgen has filed for regulatory approval of Nplate inCanada and Switzerland and these applications are currently under review.Nplate has also received orphan designation for ITP in the U.S. (2003), the EU(2005), Switzerland (2005) and Japan (2006).

Nplate is the first treatment specifically developed for ITP. It is alsobeing investigated for potential use in pediatric ITP, myelodysplasticsyndrome (MDS) and chemotherapy-induced thrombocytopenia (CIT).

Important EU Nplate Safety Information

The most common side effects are headache, fatigue, arthralgia, myalgia,injection site bruising, injection site pain, oedema peripheral, dizziness,muscle spasms, nausea, contusion, diarrhea, bone marrow disorder, influenzalike illness, insomnia and pruritus.

Reoccurrence of thrombocytopenia and bleeding after cessation of treatmentand increased bone marrow reticulin have been associated with romiplostimtreatment in the clinical trials. Thrombotic/thromboembolic complications,progression of existing haematopoietic malignancies or MyelodysplasticSyndromes (MDS), and effects on red and white blood cells are all potentialrisks associated with romiplostim treatment. As with all therapeuticproteins, patients may develop antibodies to the therapeutic protein.

Important U.S. Nplate Safety Information

Serious adverse reactions associated with Nplate in clinical studies werebone marrow reticulin deposition and worsening thrombocytopenia after Nplatediscontinuation. Additional risks include bone marrow fibrosis,thrombotic/thromboembolic complications, lack or loss of response to Nplate,and hematological malignancies and progression of malignancy in patients witha pre-existing hematological malignancy or Myelodysplastic Syndrome (MDS).Nplate is not indicated for the treatment of thrombocytopenia due to MDS orany cause of thrombocytopenia other than chronic ITP.

In the U.S. Nplate is available only through a restricted distributionprogram called Nplate(TM) NEXUS (Network of Experts Understanding andSupporting Nplate and Patients) Program.

In the placebo-controlled studies, headache was the most commonly reportedadverse drug reaction.

About Amgen

Amgen discovers, develops, manufactures and delivers innovative humantherapeutics. A biotechnology pioneer since 1980, Amgen was one of the firstcompanies to realize the new science's promise by bringing safe and effectivemedicines from lab, to manufacturing plant, to patient. Amgen therapeuticshave changed the practice of medicine, helping millions of people around theworld in the fight against cancer, kidney disorder, rheumatoid arthritis, andother serious illnesses. With a deep and broad pipeline of potential newmedicines, Amgen remains committed to advancing science to dramaticallyimprove people's lives. To learn more about our pioneering science and ourvital medicines, visit www.amgen.com.

Forward-Looking Statement

This news release contains forward-looking statements that are based onmanagement's current expectations and beliefs and are subject to a number ofrisks, uncertainties and assumptions that could cause actual results to differmaterially from those described. All statements, other than statements ofhistorical fact, are statements that could be deemed forward-lookingstatements, including estimates of revenues, operating margins, capitalexpenditures, cash, other financial metrics, expected legal, arbitration,political, regulatory or clinical results or practices, customer andprescriber patterns or practices, reimbursement activities and outcomes andother such estimates and results. Forward-looking statements involvesignificant risks and uncertainties, including those discussed below and morefully described in the Securities and Exchange Commission (SEC) reports filedby Amgen, including Amgen's most recent annual report on Form 10-K and mostrecent periodic reports on Form 10-Q and Form 8-K. Please refer to Amgen'smost recent Forms 10-K, 10-Q and 8-K for additional information on theuncertainties and risk factors related to our business. Unless otherwisenoted, Amgen is providing this information as of June 15, 2008 and expresslydisclaims any duty to update information contained in this news release.

No forward-looking statement can be guaranteed and actual results maydiffer materially from those we project. Discovery or identification of newproduct candidates or development of new indications for existing productscannot be guaranteed and movement from concept to product is uncertain;consequently, there can be no guarantee that any particular product candidateor development of a new indication for an existing product will be successfuland become a commercial product. Further, preclinical results do notguarantee safe and effective performance of product candidates in humans. Thecomplexity of the human body cannot be perfectly, or sometimes, evenadequately modeled by computer or cell culture systems or animal models. Thelength of time that it takes for us to complete clinical trials and obtainregulatory approval for product marketing has in the past varied and we expectsimilar variability in the future. We develop product candidates internallyand through licensing collaborations, partnerships and joint ventures.Product candidates that are derived from relationships may be subject todisputes between the parties or may prove to be not as effective or as safe aswe may have believed at the time of entering into such relationship. Also, weor others could identify safety, side effects or manufacturing problems withour products after they are on the market. Our business may be impacted bygovernment investigations, litigation and products liability claims. Wedepend on third parties for a significant portion of our manufacturingcapacity for the supply of certain of our current and future products andlimits on supply may constrain sales of certain of our current products andproduct candidate development.

In addition, sales of our products are affected by the reimbursementpolicies imposed by third-party payors, including governments, privateinsurance plans and managed care providers and may be affected by regulatory,clinical and guideline developments and domestic and international trendstoward managed care and healthcare cost containment as well as U.S.legislation affecting pharmaceutical pricing and reimbursement. Governmentand others' regulations and reimbursement policies may affect the development,usage and pricing of our products. In addition, we compete with othercompanies with respect to some of our marketed products as well as for thediscovery and development of new products. We believe that some of our newerproducts, product candidates or new indications for existing products, mayface competition when and as they are approved and marketed. Our products maycompete against products that have lower prices, established reimbursement,superior performance, are easier to administer, or that are otherwisecompetitive with our products. In addition, while we routinely obtain patentsfor our products and technology, the protection offered by our patents andpatent applications may be challenged, invalidated or circumvented by ourcompetitors and there can be no guarantee of our ability to obtain or maintainpatent protection for our products or product candidates. We cannot guaranteethat we will be able to produce commercially successful products or maintainthe commercial success of our existing products. Our stock price may beaffected by actual or perceived market opportunity, competitive position, andsuccess or failure of our products or product candidates. Further, thediscovery of significant problems with a product similar to one of ourproducts that implicate an entire class of products could have a materialadverse effect on sales of the affected products and on our business andresults of operations.

The scientific information discussed in this news release related to ourproduct candidates is preliminary and investigative. Such product candidatesare not approved by the U.S. Food and Drug Administration (FDA), and noconclusions can or should be drawn regarding the safety or effectiveness ofthe product candidates. Only the FDA can determine whether the productcandidates are safe and effective for the use(s) being investigated. Further,the scientific information discussed in this news release relating to newindications for our products is preliminary and investigative and is not partof the labeling approved by the FDA for the products. The products are notapproved for the investigational use(s) discussed in this news release, and noconclusions can or should be drawn regarding the safety or effectiveness ofthe products for these uses. Only the FDA can determine whether the productsare safe and effective for these uses. Healthcare professionals should referto and rely upon the FDA-approved labeling for the products, and not theinformation discussed in this news release.CONTACT: Amgen, Thousand Oaks Sabeena Ahmad, +41 41-3692-530 (EU media, oncology) Trish Hawkins, +1 805-447-5631 (U.S. media) Arvind Sood: +1 805-447-1060 (investors) (Logo: http://www.newscom.com/cgi-bin/prnh/20081015/AMGENLOGO)

SOURCE Amgen
Sponsored Post and Backlink Submission


Latest Press Release on General News

This site uses cookies to deliver our services.By using our site, you acknowledge that you have read and understand our Cookie Policy, Privacy Policy, and our Terms of Use  Ok, Got it. Close