Eteplirsen Granted FDA Accelerated Approval

The U.S. Food and Drug Administration approved Eteplirsen CAS# 1173755-55-9 (marketed as Exondys 51) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD). Exondys 51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with DMD.

“Patients with a particular type of Duchenne muscular dystrophy will now have access to an approved treatment for this rare and devastating disease,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research. “In rare diseases, new drug development is especially challenging due to the small numbers of people affected by each disease and the lack of medical understanding of many disorders. Accelerated approval makes this drug available to patients based on initial data, but we eagerly await learning more about the efficacy of this drug through a confirmatory clinical trial that the company must conduct after approval.”

DMD is a rare genetic disorder characterized by progressive muscle deterioration and weakness. It is the most common type of muscular dystrophy. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. The first symptoms are usually seen between three and five years of age, and worsen over time. The disease often occurs in people without a known family history of the condition and primarily affects boys, but in rare cases it can affect girls. DMD occurs in about one out of every 3,600 male infants worldwide. Read Article

FDA Approves Pivotal Cardiovascular Drug Vorapaxar

Vorapaxar CAS 618385-01-6 Approved by the FDAOn May 8, 2014 the U.S. Food and Drug Administration approved the formidable cardiovascular drug Vorapaxar. Known as the brand name Zontivity, which is marketed by Merck, this pivotal medication has proven to be outstanding in clinical trials. In three short years of study Vorapaxar demonstrated its ability to successfully reduce the rate of death in over 17 percent of patients who had suffered cardiovascular trauma. Vorapaxar also lowered the risk of cardiovascular events and myocardial infractions in patients who were diagnosed with peripheral arterial disease. The current best in care treatment for patients who have survived traumatic cardiovascular events, such as a heart attack, is not effectual for every patient. Despite standard treatment with medications like Clopidogrel and aspirin, the need for adjunctive medications is growing increasingly common among the patients who have experienced cardiovascular trauma. The concomitant administration of Vorapaxar alongside aspirin and statins is an effective way to sizably lower the risk of additional cardiac events in this patient population.

Vorapaxar is a first-in-class inhibitor of the protease-activated receptor and is deemed to be an efficacious treatment option for patients who have endured a prior heart attack or have peripheral arterial disease. Data submitted to the FDA from Merck also offered encouraging statistics regarding a notable decrease in stroke risk for patients who were administered Vorapaxar {Zontivity}. At the conclusion of three years patients who were given Vorapaxar demonstrated a decline of stroke risk from 9.5 percent to 7.9 percent. While there is a black-box warning for Vorapaxar due to the potential for fatal bleeding episodes, the overall cardiac benefit of this potent drug is apparent for the almost 200,000 Americans who suffer from recurrent heart attacks after their first cardiovascular event.

 

Vorapaxar

Data disseminated from the TRA 2°P TIMI-50 study, which involved 499 patients overwhelmingly met the primary end point. Roughly 70 percent of patients enrolled in the aforementioned study had a prior myocardial infarction, and the primary goals from the study were to significantly reduce the risk of cardiovascular death and reduce repeated myocardial infarction. The patients in the TRA 2°P TIMI-50 study who were treated with 2.5 milligrams of Vorapaxar daily experienced a 13 percent reduction in the primary study end point. The study panel, which included experts from the Vanderbilt University School of Medicine, advised that the benefits of Vorapaxar outweighed the negative effect of excessive bleeding, but expressed that patients who had experienced a prior TIA or stroke should refrain from using this medication.

LGM Pharma provides Vorapaxar API, CAS# 618385-01-6 for research and development purposes. Clients can be assured of quality API products and continuous support throughout the R&D process.

 

Products currently covered by valid US Patents are offered for R&D use in accordance with 35 USC 271(e)+A13(1). Any patent infringement and resulting liability is solely at buyer risk.

FDA Approves Bosutinib for Chronic Myelogenous Leukemia

Bosutinib CAS# 380843-75-4The U.S. Food and Drug Administration today approved Bosutinib CAS# 380843-75-4, marketed under the trade name Bosulif, to treat chronic myelogenous leukemia (CML), a blood and bone marrow disease that usually affects older adults.

An estimated 5,430 men and women will be diagnosed with CML in 2012. Most people with CML have a genetic mutation, called the Philadelphia chromosome, which causes the bone marrow to make an enzyme called tyrosine kinase. This enzyme triggers the development of too many abnormal and unhealthy white blood cells called granulocytes. Granulocytes fight infection.

Bosulif is intended for patients with chronic, accelerated or blast phase Philadelphia chromosome positive CML who are resistant to or who cannot tolerate other therapies, including imatinib. Bosulif works by blocking the signal of the tyrosine kinase that promotes the development of abnormal and unhealthy granulocytes.

“With the approval of tyrosine kinase inhibitors, we are seeing improvements in the treatment of CML based on a better understanding of the molecular basis of the disease,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “These improvements have been observed in chronic and accelerated phases of CML.”

Other drugs recently approved by FDA to treat various forms of CML include imatinib (2001), dasatinib (2006) and nilotinib (2007).

The safety and effectiveness of Bosulif was evaluated in a single clinical trial that enrolled 546 adult patients who had chronic, accelerated or blast phase CML. All patients had disease that progressed after treatment with imatinib or imatinib followed by dasatinib and/or nilotinib, or who could not tolerate the side effects of prior therapy. All patients in the trial were treated with Bosulif.

In patients with chronic phase CML, efficacy was determined by the number of patients who experienced a major cytogenetic response (MCyR) within the first 24 weeks of treatment. Results showed 34 percent of patients who had been previously treated with imatinib achieved MCyR after 24 weeks. Of the patients who achieved MCyR at any time, 52.8 percent had their response last at least 18 months. Among patients previously treated with imatinib followed by dasatinib and/or nilotinib, about 27 percent achieved MCyR within the first 24 weeks of treatment. Of those who achieved MCyR at any time, 51.4 percent had their MCyR last at least nine months.

In patients with accelerated CML previously treated with at least imatinib, 33 percent had their blood counts that returned to normal range (complete hematologic response) and 55 percent achieved normal blood counts with no evidence of leukemia (overall hematologic response) within the first 48 weeks of treatment. Meanwhile, 15 percent and 28 percent of patients with blast phase CML achieved complete hematologic response and overall hematologic response, respectively.

The most common side effects observed in those receiving Bosulif were diarrhea, nausea, a low level of platelets in the blood (thrombocytopenia), vomiting, abdominal pain, rash, low red blood cell count (anemia), fever and fatigue.

Bosulif is marketed by New York City-based Pfizer.

Source URL: http://www.fiercebiotech.com/press-releases/fda-approves-new-orphan-drug-chronic-myelogenous-leukemia

 

Products currently covered by valid US Patents are offered for R&D use in accordance with 35 USC 271(e)+A13(1). Any patent infringement and resulting liability is solely at buyer risk.

Pixantrone Maleate as a New Treatment for Patients with Non-Hodgkin’s Lymphoma

Pixantrone MaleatePixantrone maleate, CAS Number 146675-97-8, is an anthracenedione anthracycline derivative, developed by Cell Therapeutics Incorporated. Pixantrone maleate is used for treatment of patients with non-Hodgkin’s lymphoma. LGM Pharma is a provider of pixantrone maleate for research and development purposes.

As a promising treatment for aggressive non-Hodgkin’s lymphoma, pixantrone maleate is well tolerated when substituted for anthracyclines in combination regimens, and shows comparable rates of complete remission. Pixantrone is similar in structure to the anthracenedione mitoxantrone, however it lacks the 5,8-dihydroxy substitution groups, which is found in mitoxanthrone. Mitoxanthrone was shown in studies to induce the rates of patient cardiotoxicity in patients. A trial, deemed PIX301 phase III, used pixantrone maleate as a single-agent for patients with relapsed or refractory aggressive non-Hodgkin’s lymphoma. For this trial randomized patients received either pixantrone or another single agent of the investigators’ choice. Positive results were found, showing the rate of confirmed and unconfirmed remissions in patients treated with pixantrone was significantly higher than in those patients who received other agents. The overall response rate and progression-free survival in patients in the trial were also demonstrated in the PIX301 phase lll trial.  Another study, phase III PIX302, treated patients with indolent non-Hodgkin’s lymphomas, and combined pixantrone and rituximab in treatment. Trials found this combination to be superior to rituximab used alone in patients with relapsed or refractory disease. Evidence in all trials also shows that pixantrone maleate is well tolerated when substituted for anthracyclines in combination regimens for aggressive non-Hodgkin’s lymphoma .

non-Hodgkin's Lymphoma

Photo courtesy of http://www.cancer.gov

The United States Food and Drug Administration is in the process of considering pixantrone for use in adult patients with relapsed or refractory aggressive non-Hodgkin’s lymphoma, as well as and indolent non-Hodgkin’s lymphoma, on a fast-track basis. LGM Pharma can work with its clients through all research stages, clinical trials, development phases, regulatory documentation submission and up till commercialization of the pixantrone maleate product. Click here to inquire about Pixantrone Maleate CAS# 146675-97-8.

Products currently covered by valid US Patents are offered for R&D use in accordance with 35 USC 271(e)+A13(1). Any patent infringement and resulting liability is solely at buyer risk.

Liraglutide diabetes control is proven and leads to weight loss

Liraglutide Diabetes Control

LiraglutideLiraglutide is a newly developed drug that is used to control the levels of blood glucose in patients suffering from type 2 Diabetes. In January 2010, the US Food and Drug Administration approved this drug for the treatment of patients with diabetes. Liraglutide was approved by the European Medicines Agency (EMEA) in 2009. Researchers have confirmed the efficacy of Liraglutide in lowering weight and blood sugar levels of patients suffering with early type 2 Diabetes.

Usefulness of Liraglutide

Just like Byetta, Liraglutide is an analog of a glucagon like peptide known as GLP-1 that can stimulate the pancreas to expand beta cells. These beta cells produce insulin. Recent researches have shown that unlike Byetta, that causes serious side effects of pancreatitis, Liraglutide is a safer medicine.

Liraglutide helps patients by controlling hyperglycemia and by increasing insulin secretion in diabetic patients. This drug efficiently reduces the secretion of prandial glucagon, and helps patients to digest their meal without causing any abrupt increase in blood sugar. This drug is safe against hyperglycemia risks because it stimulates insulin secretion in the pancreas only when the concentration of blood sugar is abnormally higher. Recent scientific research has shown that Liraglutide can increase the number of insulin-producing beta cells in the pancreas by reducing the apoptosis of beta cells. The drug is efficient in reducing triglyceride levels as well as controlling Type 2 Diabetes patients’ appetites, leading to significant weight loss.

Recent Studies and results

During a study on mice in 2009, researchers observed that Liraglutide may cause malignant C-cell carcinoma and thyroid C-cell focal hyperplasia that may result in thyroid cancer in mice and rats. However, studies have established that Liraglutide is safe for humans. In January 2010, Liraglutide was approved by the FDA for administering to adult diabetic patients.

Researchers have also established that Liraglutide is safe against any risks of hyperglycemia because it remains inactive during periods when blood sugar concentrations are normal. Thus, while Liraglutide can be efficiently used to reduce the levels of blood glucose, it cannot cause any harm by lowering the glucose concentration below normal levels.

Availability of Liraglutide

Liraglutide CAS# 204656-20-2, as well as many other Anti-Diabetic products are available from LGM Pharma. Inquire about prices, availability, delivery, purity and more.

*Products currently covered by valid US Patents are offered for R&D use in accordance with 35 USC 271(e)+A13(1). Any patent infringement and resulting liability is solely at buyer risk.