New Study Shows Nplate(R) Significantly Reduces Splenectomy Rate and Treatment Failure

Amgen Inc.Amgen Inc. (Nasdaq: AMGN) released the results of a new study comparing Nplate(R) (romiplostim) to the medical standard of care (SOC) in non-splenectomised adult patients with chronic immune thrombocytopenic purpura (ITP). Chronic ITP is a serious autoimmune disorder characterised by low platelet counts in the blood (thrombocytopenia), which can lead to serious bleeding events. The study results show Nplate significantly reduced the incidences of splenectomy and treatment failures in non-splenectomised adult patients with chronic ITP when compared to medical SOC. The results were presented today as an oral presentation at the 14th congress of the European Hematology Association (EHA abstract #1672).

"In this study, patients receiving Nplate experienced significant clinical efficacy benefits, including a reduction in bleeding events, compared to the standard of care," said Dr. Mathias Rummel, head of hematology at the Hospital of the Justus-Liebig University, Giessen, Germany. "Nplate is a unique treatment option that can help us better manage patients with chronic ITP. It addresses an unmet medical need for our patients as it increases platelet production and avoids immune suppression."

The study results show that only 8 percent of Nplate patients (13/157) underwent splenectomy or discontinued the study prior to reporting a splenectomy compared with 35 percent of patients (27/77) in the SOC group. Furthermore, 12 percent of Nplate patients (19/157) experienced treatment failure or discontinued the study compared with 27 percent of the SOC patients (21/77). Treatment failures were defined as patients having platelet counts less than or equal to 20,000 platelets per microliter for four consecutive weeks at the highest recommended dose and schedule, a major bleeding event, and/or a change in therapy due to intolerable side effects or bleeding symptoms. Patients who changed their therapy to splenectomy due to intolerable side-effects or bleeding symptoms were counted as both treatment failures and splenectomies.

A secondary analysis excluding patients who discontinued the study showed a similar trend in the reduction in splenectomy and treatment failure in the Nplate group compared to the SOC group. Only 1 percent of Nplate patients (2/157) underwent a splenectomy compared with 19 percent of SOC patients (15/77). Additionally, 5 percent of Nplate patients (8/157) experienced treatment failure compared to 12 percent of SOC patients (9/77).

The study also showed that the safety profile was comparable between the Nplate group and the group receiving the SOC. The safety analyses included all patients who received greater than or equal to 1 dose of Nplate or one type of SOC for ITP. Bleeding events with grade greater than or equal to 3 severity were reported by 8 percent of patients (6/75) in the SOC group, compared with 3 percent in the Nplate group (5/154).

About the Study
In total, 234 patients enrolled in this study, which assessed the efficacy and safety of Nplate compared to the medical SOC in adult patients with chronic ITP. SOC treatments were prescribed by the investigator according to standard institutional practices or therapeutic guidelines; the only treatments not allowed were investigational agents (rituximab was allowed) or other thrombopoietic agents.

Adverse events (AEs) were experienced by 92 percent of patients receiving the SOC (69/75) and by 95 percent of patients (146/154) receiving Nplate. The most common AEs in the SOC group were epistaxis (23 percent), nasopharyngitis (19 percent), and contusion (19 percent); in the Nplate group the most common AEs were headache (35 percent), fatigue (27 percent), and nasopharyngitis (23 percent). Treatment-related serious AEs were reported by 8 percent of SOC (6/75) and 5 percent of Nplate patients (7/154).

About Adult Chronic ITP
In patients with chronic ITP, platelets - or blood elements needed to prevent bleeding - are destroyed by the patient's own immune system. Low platelet counts leave adult ITP patients open to sudden serious bleeding events. The risk for serious bleeding events can increase when platelet counts drop to less than 30,000 platelets per microlitre; normal counts range from 150,000 to 400,000 platelets per microlitre. ITP has historically been considered a disease of platelet destruction although recent data suggest that the body's natural platelet production processes in chronic ITP are unable to compensate for low levels of platelets in the blood. Increasing the rate of platelet production may address low platelet levels associated with ITP.

Currently available treatments (e.g., corticosteroids, immunoglobulins and others) have limited application due to poor tolerability or transient effects. Surgical therapy (removal of the spleen) is also available to adult patients with chronic ITP, but does not work in all cases. Currently, there are approximately 140,000 treated chronic ITP patients in Europe (EU) and the United States (U.S.). Chronic ITP affects about twice as many adult women as men.

About Nplate
In Europe, Nplate is indicated for the treatment of splenectomised adult chronic immune (idiopathic) thrombocytopenic purpura (ITP) patients who are refractory to other treatments (e.g. corticosteroids, immunoglobulins). Nplate may be considered as a second-line treatment for adult non-splenectomised ITP patients for whom surgery is contra-indicated.

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

Nplate was the first platelet producer approved for chronic ITP by the regulatory bodies in Australia, the EU, Canada, and the U.S., and is under review in Switzerland. Nplate also has received orphan designation for chronic ITP in the U.S. (2003), the EU (2005), Switzerland (2005) and Japan (2006).

Nplate is the first treatment specifically developed for chronic ITP. It is also being investigated for potential use in paediatric ITP, myelodysplastic syndromes (MDS), and chemotherapy-induced thrombocytopenia (CIT).

About Amgen
Amgen discovers, develops, manufactures and delivers innovative human therapeutics. A biotechnology pioneer since 1980, Amgen was one of the first companies to realize the new science's promise by bringing safe and effective medicines from lab, to manufacturing plant, to patient. Amgen therapeutics have changed the practice of medicine, helping millions of people around the world in the fight against cancer, kidney disorder, rheumatoid arthritis, and other serious illnesses. With a deep and broad pipeline of potential new medicines, Amgen remains committed to advancing science to dramatically improve people's lives. To learn more about our pioneering science and our vital medicines, visit www.amgen.com.

Most Popular Now

Sanofi to acquire Ablynx for €3.9 Billion

Sanofi and Ablynx, a biopharmaceutical company engaged in the discovery and development of Nanobodies®, entered into a definitive agreement under which Sanofi will offer ...

In wine, there's health: Low levels of alcohol goo…

While a couple of glasses of wine can help clear the mind after a busy day, new research shows that it may actually help clean the mind as well. The new study, which appe...

Repurposed drug found to be effective against Zika…

In both cell cultures and mouse models, a drug used to treat Hepatitis C effectively protected and rescued neural cells infected by the Zika virus - and blocked transmiss...

More stroke patients may receive crucial treatment…

More patients could be eligible for critical treatments to remove or dissolve blood clots that cause strokes, according to a new treatment guideline issued by the America...

Interim publications of randomized trials make new…

Early results from randomized trials are sometimes published before the trial is completed. The results of such interim publications may generate a great deal of interest...

Drug trial protocol redactions by industry sponsor…

New research published by the Journal of the Royal Society of Medicine exposes the extent of redactions in protocols for industry-sponsored randomised drug trials. Trial ...

Advanced Accelerator Applications receives FDA ap…

Novartis AG (NYSE: NVS) announced that Advanced Accelerator Applications, a subsidiary of Novartis Groupe S.A., has received US Food and Drug Administration (FDA) approva...

Blood vessel-on-a-chips show anti-cancer drug effe…

Researchers at the Institute of Industrial Science (IIS), the University of Tokyo, CNRS and INSERM, report a new organ-on-a-chip technology for the study of blood vessel ...

Roche reports good results in 2017

In 2017, Group sales rose 5% to CHF 53.3 billion. Core operating profit grew 3% and Core EPS increased 5%, reflecting the good underlying business performance. On an IFRS...

FDA approves new treatment for certain digestive t…

The U.S. Food and Drug Administration today approved Lutathera (lutetium Lu 177 dotatate) for the treatment of a type of cancer that affects the pancreas or gastrointesti...

Cells of 3 advanced cancers die with drug-like com…

Researchers at Southern Methodist University have discovered three drug-like compounds that successfully reverse chemotherapy failure in three of the most commonly aggres...

Pfizer announces positive top-line results for pot…

Pfizer Inc. has announced that REFLECTIONS B3281006, a comparative safety and efficacy study of PF-05280586 versus MabThera® (rituximab-EU), met its primary endpoint. PF-...

Pharmaceutical Companies

[ A | B | C | D | E | F | G | H | I | J | K | L | M | N | O | P | Q | R | S | T | U | V | W | X | Z ]