EMA accepts the application for authorization of Bavencio for renal cell carcinoma

The European Medicines Agency (EMA) accepted for consideration a authorization application from Merck KGaA for Bavencio (avelumab) for the treatment of patients with advanced renal cell carcinoma (CRP).

The regulator is considering the application for the use of Bavencio in combination with Inlyta (axitinib), which was submitted on the results of Phase III pivotal clinical trial (CI) JAVELIN Renal 101.

JAVELIN includes more than 30 research programs; more than 9 thousand patients with more than 15 different types of tumors take part in researches.

In addition to RCC, Bavencio therapy for breast cancer, gastric cancer / gastroesophageal cancer, head and neck cancer, Merkel carcinoma, non-small cell lung cancer and urothelial carcinoma is being investigated.

The CI results were published in the New England Journal of Medicine in February 2019.

The FDA also accepted an application for this combination of drugs for distributed RCC for an accelerated review.

CRP is the most common form of kidney cancer, which occurs in 2–3% of adults with this disease.

Link: pharmvestnik.ru

Tecentriq was approved in the US for the treatment of breast cancer

Roche’s immunotherapy drug Tecentriq was approved in the United States for treatment of patients suffering from triple negative breast cancer (TNBC), which is a progress in fight with this aggressive disease.

The FDA approved Tecentriq in combination with a chemotherapeutic drug Abraxane for the treatment of inoperable locally advanced or metastatic TNMP in patients with PD-L1 protein expression.

In a study conducted by Roche, approximately 40% of patients had tumors with a high level of PD-L1 expression.

The FDA approved this combination of drugs based on clinical study showed a slower progression of the disease by 7.4 months (median value) compared with 4.8 months when chemotherapy was used alone.

Roche is still awaiting data on overall survival and its specialists noted that positive opinion of a regulator may depend on results of further supporting studies.

According to Roche Medical Director Sandra Horning, the Tecentriq combination is one of the first cancer immunotherapy regimens approved for breast cancer and symbolizes significant progress in understanding this disease.

Link: pharmvestnik.ru

For clinical trials, ‘convenience’ services to be a standard offering in 2019

The industry’s pledge to improving the patient experience drove significant change in 2018 and will continue to do so this year, with so-called “convenience” services to become standard, says Greenphire CCO, who outlines several other trends to watch.

The clinical trial sector’s overall commitment to improving the patient experience was perhaps the most significant positive change seen over the past year. High dropout rates across all trials – around 30%, according to studies – and 85% of trials failing to enroll enough patients has driven this paradigm shift.

“Improving these numbers is paramount to driving better patient outcomes, and the key is keeping participants engaged throughout the study and removing barriers to participation,” said Wayne Baker, chief commercial officer, Greenphire, a clinical trial payments solution company.

Removing logistical barriers

A survey from the National Institutes of Health cited a “lack of transportation” as one such barrier, particularly among certain participant demographics. As a result, several companies have partnered with ride-sharing companies, such as Uber and Lyft, to remove the logistical burden of organization and paying for transportation.

Moving into 2019, Baker predicts these types of “convenience” services will become a standard offering in clinical research. The industry also will begin to see a shift toward more site-less and/or hybrid trials, he said.

“With the increasing use of technology in health care, participants can communicate with study staff remotely and receive their medications in the mail,” Baker explained, “while mobile health technologies like Fitbit, Apple Watch, Telcare, and others allow participants to transmit real-time data directly to study sites.”

As this trend matures, Baker said sites will look for new ways to engage and retain participants for the duration of a study.

Changing data privacy legislation

Regulations such as the General Data Protection Regulation (GDPR ), the California Consumer Privacy Act , the Physician Payments Sunshine Act , and others, “will continue to influence how clinical trial sponsors and sites capture and store participant data, and what they can do with it,” noted Baker.

Ultimately, changing regulations will drive sponsors, sites, and vendors to update and strengthen data system architectures – and will require heightened due diligence to ensure compliance, he said.

“Like to 2018, regulatory changes, and the disparate way that regulations are enforced per state and per country, will continue to be a challenge for sponsors and sites conducting global clinical trials,” Baker added.

“Data privacy will be more scrutinized; therefore, it is crucial to ensure the proper security measures are in place to meet regulatory requirements around the world.”

Prioritizing payments

Pharma companies have been under mounting pressure to increase transparency as it pertains to how funds are dispersed – with some $125-$160bn spent annually on global R&D, according to Greenphire.

Subsequently, Baker said sponsors and sites will prioritize clinical trial payments as a budget item in 2019, “with an increased emphasis on improving financial control and overall visibility.”

“By standardizing and centralizing global investigator payments, sponsors can better ensure that clinical trial-related payments are executed consistently, accurately and in compliance with financial regulations around the world, benefiting sites and sponsors,” he added.

Using business intelligence

Coming closer to a reality in 2019, is the use of business intelligence to support clinical trial feasibility, Baker said, as the need for better insight into clinical trial data trends – which can support feasibility teams – grows.

“Through the use of technology solutions that aggregate and use data in a smart/dynamic way that can be shared with other interconnected software systems, sponsors will have far more accurate business intelligence for enhanced forecasting capabilities that can be used as a baseline for future studies and in support of feasibility,” Baker added.

Link: www.outsourcing-pharma.com

In the US, approved a drug for the treatment of rare blood diseases

Sanofi received a Marketing Authorization from US regulatory authorities for caplacizumab (caplacizumab) for the treatment of thrombotic thrombocytopenic purpura. This is quite a serious and rare disease. Such therapy is registered only for adult patients.

This decision was made after studying the results of a clinical study that was conducted with 145 patients.

Clinical trial showed the superiority of new drug to placebo.

Kaplacizumab is a single immunoglobulin variable domain that blocks the interaction between von Willebrand factor multimers and platelets.

The von Willebrand factor is a blood plasma glycoprotein that secures the attachment of platelets to the site of the damaged vessel.

In Europe, caplacizumab was approved in the autumn of 2019. This is the development of the Belgian company Ablynx.

After its acquisition by Sanofi for 3.9 billion euros, the rights were transferred to the French pharmaceutical manufacturer.

Link: chemrar.ru

Scientific Research Institute of Highly Pure Biopreparations Resumes Production of Antioxidant

The Federal State Unitary Enterprise “State Scientific Research Institute of Highly Pure Biopreparations” FMBA decided to return to the market the original drug Rexod® (INN: superoxide dismutase), which was developed and produced by the Institute.

The drug is available in a new dosage form – solution for injection.

Superoxide dismutase is an endogenous acceptor of free oxygen radicals. Because of their excessive accumulation in the cells, pathological processes develop, and the current removes free radicals and prevents the formation of new hydroxyl and singlet radicals that are dangerous for the body.

An important pharmacological action of the drug is prevention of accumulation in the focus of inflammation of neutrophils, causing the destruction of nearby tissues.

The developers have conducted a number of studies and plan to expand the scope of the drug for the treatment of complications of radiation therapy of cancer, as well as in lateral amniotrophic sclerosis and in resuscitation practice.

Currently, the only indicator is the comprehensive prevention of the development of intraoperative complications in endoprosthetics of large joints using bone cement.

Link: pharmvestnik.ru

The medicine created in the Russian Federation has passed clinical trials as a remedy for tick-borne encephalitis

Antiviral drug triazavirin was developed by researchers of the Ural Branch of the Russian Academy of Sciences and the Ural Federal University.

Employees of the Krasnoyarsk State Medical University conducted clinical trials of antiviral drug triazavirin, developed by Ural researchers, and confirmed its effectiveness for tick-borne viral encephalitis (EEC). It was reported by the press service of the Ministry of Science and Higher Education of the Russian Federation.

According to statistics, the Krasnoyarsk Territory is considered the largest natural foci of tick-borne encephalitis in Russia. Over the past years, the incidence in the region has exceeded the average Russian indicators by 9.5 times. Despite the use of specific antiviral and immunotropic drugs, such as RNA-aza, ribavirin, panavir, ferrovir, new cases of disability and death of inhabitants of the region infected with encephalitis are recorded annually.

“Triazavirin, developed by researchers of the Ural Branch of the Russian Academy of Sciences (Ural Branch of the Russian Academy of Sciences) and the Ural Federal University (Ural Federal University, Yekaterinburg), proved to be an effective remedy against tick-borne encephalitis (EHE). Clinical trials conducted by Krasnoyarsk State Medical University “- said in a statement.

In search of effective treatments for tick-borne viral encephalitis, university staff decided to study the properties of triazavirin. This drug, which has been developed since the late 1980s, entered the pharmacy chain in 2014 and proved itself as a powerful tool against influenza viruses (including avian and swine), bronchial asthma, and also rubella, measles, and herpes. In addition, triazavirin has been shown to be effective against Ebola virus, Marburg hemorrhagic fever, and West Nile fever. The main mechanism of drug action is the blockade of viral RNA synthesis.

The study, conducted in the Krasnoyarsk Interdistrict Clinical Emergency Hospital, involved 73 patients with febrile forms of tick-borne viral encephalitis. The inclusion of triazavirin in the treatment regimen, as the scientists note, was accompanied by a decrease in all clinical manifestations of this disease: fever stopped by 2.3 times, intoxication syndrome by 1.8 times, catarrhal phenomena by 1.7 times faster.

“The results of the study allow us to assign triazavirin, taking into account the mechanism of its action in tick-borne viral encephalitis, to the group of etiotropic agents with good efficacy and tolerability,” the report says. In addition to the Krasnoyarsk Territory, the drug may be in demand in other regions – in the Urals, in Western and Eastern Siberia, where tick-borne encephalitis is an endemic disease.

Link: tass.ru

UK and Russia sign MoU on Regulatory Cooperation on Medicines

Medicines regulators of the UK and Russian set out key principles to support their collaboration on improving public health.

The Medicines and Healthcare products Regulatory Agency (MHRA) has signed a Memorandum of Understanding (MoU) with its Russian counterpart; the State Institute of Drugs and Good Practices of the Ministry of Industry and Trade of the Russian Federation.

This agreement will increase collaboration between the two bodies and further enhance cooperation in the field of regulation of medicines.

The MoU is designed to establish mechanisms of cooperation which will facilitate:

  • the exchange of information on medicines regulations
  • the exchange of safety information
  • cooperation on Good Manufacturing Practice (GMP) inspections
  • collaboration on enforcement activities

Dr Sam Atkinson, MHRA’s Director of the Inspection, Enforcement and Standards Division, said:

“Working in an increasingly global environment, the sharing of intelligence between medicines regulators is the cornerstone of protecting the health system worldwide.”

“Formalising our already close working relationships with international counterparts helps facilitate this exchange of information.”

“This makes it easier for us to track down criminals, promote good practices internationally, and identify emerging safety trends to protect public health.”

Link: www.gov.uk

New results of the Phase III clinical trial of Tafamidis

In the section of the newest clinical trials of the 22nd annual scientific conference of American society in heart failure there were the results of the sensitivity and post-hoc analysis. New analysis of death due to any cause has shown the benefits of Tafamidis in all subgroups.

For example, among patients with cardiomyopathy due to the wild-type  transthyretin and the hereditary form of the disease, the risk of death decreased by 29% and 31%, respectively. Pfizer Inc. announced that additional sensitivity analysis and post-hoc analysis that were conducted during the Phase III clinical trial of Tapamidis for the treatment of transthyretin family amyloid cardiomyopathy (Att-ACT) provided additional information about the action of the drug in subgroups of patients with cardiomyopathy due to the wild-type transthyretin and the hereditary form of the disease, and subgroups of patients with HSN I, II, and III FC NYHA1.

Tafamidis is the single experimental drug that has successfully passed the Phase III clinical trial, which evaluates its safety and efficacy for the treatment of the disease.

ATRT-CMC is a rare and poor diagnosed fatal disease associated with progressive heart failure for which there is no approved pharmacological therapy.

The primary results of the study were presented on August 27, 2018 at the Congress of the European Society of ECC 2018 cardiologists in Munich (Germany) and were simultaneously published on the Internet in the Nejm (“New Joggy Medical Medical Journal”).

Additional primary results showed that in comparing with placebo Tafamidis during 30 months greatly reduces the combination of death due to any cause and the frequency of hospitalization associated with cardiovascular manifestations of diseases, in patients with “wild type” ATRT-CMC and a hereditary form of the disease.

In addition, in comparing with the placebo, Tafamidis reduced the risk of death due to any cause in all subgroups (wild type disease, hereditary disease and functional classes NYHA I, II and III). “These additional data also confirm that Tafamidis is a promising cure for treatment of “wild type” ATRT-KM or hereditary disease,” Brenda Cuperston, the Senior Vice President and the head of the Development Defense, the main specialist of the drug department for the treatment of orphan diseases and the development of global products of Pfizer, said. – We are looking forward to get more information  from analysis of this study and continue to work with regulatory authorities of different countries to make this drug available for patients. ”

“Having received statistically significant results of the primary analysis of the Phase III clinical trial of Att-ACT, we sought to go deep into the efficacy of Tafamidis in these key subgroups of patients,Mathew S. Maurer said, Head Doctor of the Cathedral of the Center for the Study of Hypertrophic Cardiomyopathy at the Presbyterian Hospital in New York / the Clinic of the Columbia University, presenting the results of the Att-ACT study at the annual HFSA 2018 conference. – The additional analysis confirm the results presented at the ESC 2018 congress and published several weeks ago in the New Jognog Journal of Medicine, which emphasizes the importance of raising awareness about this potentially death disease and the importance of diagnosing the disease at earlier stage. ”

Taking into account severity of the disease and the lack of alternatives for the treatment, Pfizer approved advanced access to Tafamidis for the patients suffering from ATTR-CMC, which can benefit from the treatment before approval of the drug by supervisory authorities.

Access to such programs may differ depending on the country; health care providers are encouraged to contact the local Medical Department of Pfizer for more information.

Interested patients suffering from ATTR-CMC should contact with their doctors to discuss the expediency of treating by Tafamidis.

In 2012, Tafamidis received the status of an orphan drug for the treatment of ATTR-CMC in both EU and the United States, as well as in 2018 in Japan. In June 2017 and in May 2018, accordingly, the       Food and Drug Administration (FDA) decided to introduce an accelerated application procedure for the drug with the status of fundamentally new, breakthrough drug for the ATTR-CMP therapy.

In 2017, Tafamidis (Vyndaqel) was approved in the Russian Federation. This is the first and the single drug in Russia with an approved indication “for the treatment of transthyretin amyloidosis in adults with clinically apparent polyneuropathy in order to delay the development of disorders in peripheral nerves.”

Link: www.labclinpharm.ru

Statins may suspend the development of diabetic retinopathy

According to experts from Chang Gun Memorial Hospital in Taiwan, diabetic patients taking statins have a reduced risk of diabetic retinopathy.

The researchers, based on information from the National Taiwan Health Insurance Policy Database, undertook a comparative analysis of patients with type 2 diabetes and high cholesterol, among whom about 19,000 patients took statins, the same amount didn’t take statins. Monitoring during 7 years found out that among participants who took statins diabetic retinopathy appeared in 2004 patients (10.6%), while in the group who did not take these drugs, the disease developed in 2269 participants (12%).

The experts also noted that the use of statins was associated not only with a lower demand in invasive treatment of eye diseases, but also with a reduced risk of diabetic retinopathy.

However, according to Dr. Seth Martin, a Johns Hopkins Medicine scientist, the results of this study require careful analysis and confirmation. He notes that the main reason for taking statins is to reduce the risk of cardiovascular diseases, and the positive impact of these drugs on diabetic patient vision is an additional benefit.

Link: medvestnik.ru

Valenta Farm presents an innovative formulation of a benzodiazepine drug

A new unique formulation of Fenazepam® developed by Valenta Pharm appeared in the drugstores of our country at the beginning of December. It is Phenazepam® orally dispersible tablets 1mg N10.

Innovative dosage form is a result of the seven-year efforts made by the Company, as well as significant investment in the development and research. Phenazepam® is a unique benzodiazepine drug, which hasn’t any analogues on the Russian market.

Consumer characteristics of the new dosage form are:

– additional control of course therapy;
– the best subjective tolerance of the therapy;
– it isn’t necessary to swallow and drink water, the tablet dissolves in the mouth;
– provides a more rapid achievement of drug maximum concentration in blood: up to 29% faster compared with the traditional tablet dosage form.

The drug is produced by new technology in accordance with Good manufacturing practice (GMP) at the modern high-tech research and production complex of Valenta Ph. JSC.

“The introduction in standard practice of orally dispersible dosage forms available for use in various branches of medicine is a very promising way for increasing the therapy effectiveness and optimizing the treatment. In psychiatry the main advantage of CCT is a possible fast therapeutic effect that is comparable in strength to the effect of the oral dosage form, as well as a significant increase of patient compliance and the early adherence to the therapy. The creation of new orally dispersible dosage forms of anxiolytic drugs intended for rapid relief of acute manifestations of anxiety syndrome is of particular concern ”- Sergey Nikolayevich Mosolov, professor, doctor of medical sciences, Honoured scientist of the Russian Federation, head of the Psychiatric Research Institute of Psychiatry said.

Link: chemrar.ru