All posts by Web Team

The exceptional or rare diseases are expected to affect 30 million people in the European Union and approximately the same number in the United States, each disease independently concerns a limited number of patients.

The European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA) have new collaboration on exceptional diseases to share experiences and best practices on each other’s regulatory approach to the development of medicines for these diseases.

The international alliance in this area is mainly important to make sure that the limited number of studies that can be conducted to benefit all patients.

The agencies will exchange information on various aspects of the development and scientific assessment of medicines for rare diseases such as:

• Plan of clinical trials in small populations and the use of statistical analysis methods;
• Substantiation of trial endpoints such as outcomes of a trial
• Preclinical proof to carry development programs
• EMA’s conditional marketing authorization and FDA’s accelerated approval
• Risk management strategies for long-term safety issues with medicines for rare diseases.

The existing EMA/FDA alliance discusses issues like:

• Patient engagement
• Bio similar
• Orphan medicines
• Medicines to treat cancer
• Medicines for children
• Pharmaco care

The first meeting of the rare diseases alliance took place by teleconference on 23 September 2016. The alliance will at first meet once a month via teleconference and will be chaired jointly by FDA and EMA.

Date: 26/09/2016

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/09/news_detail_002609.jsp&mid=WC0b01ac058004d5c1

Plasma-derived medicines are prepared from human blood. They are used to treat and prevent serious diseases and include coagulation factors and immunoglobulin. Urine-derived products are made from pooled human urine and include certain hormone-based treatments and urokinase products (medicines used to break up blood clots.

Basic Evaluations conceded out by the European Medicines Agency (EMA) and proficient establishment in the EU Member States have confirmed that there is no increased risk of contamination with the Zika virus for patients who take plasma-derived or urine-derived medicines.

EU regulators sought assurance that there is no risk of the virus contaminating the final product and affecting the patients taking it if the plasma or urine came from donors who had constricted the Zika virus.

EMA’s Committee for Medicinal Products for Human Use (CHMP) has addressed the potential risk from Zika virus for plasma-derived medicinal products. The CMDh has corresponding the assessment by EU Member States on the potential risk from Zika virus for urine-derived medicinal products.

The CHMP concluded at its meeting last week that the manufacturing processes used for plasma-derived products, including for example the solvent method to inactivate viruses, pasteurization and virus filtration inactivate or remove the Zika virus from the finished product.

Relating to urine-derived products, the CMDh concluded that the manufacturing processes for these products contain balancing steps with inactivation ability for enveloped viruses, which are adequate for Zika virus safety of these products.

Date: 21/09/2016

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/09/news_detail_002606.jsp&mid=WC0b01ac058004d5c1

The European Medicines Agency (EMA) has suggested conceding a marketing endorsement in the European Union (EU) for Ibrance for the treatment of women metastatic breast cancer.

This treatment will be effective for cancer that is hormone receptor (HR) positive and human epidermal growth factor receptor 2 (HER2) negative. Hormone receptor positive breast cancer accounts for 65% of tumors in women aged 35 to 65 years and 82% of tumors in women older than 65 years.

Breast cancer is the most common cancer in women globally, with almost 1.7 million new cases diagnosed in 2012. In Europe, there were probably 464,000 new cases of breast cancer in 2012 and an anticipated 131,000 deaths from the disease.

The commendation from EMA’s Committee for Medicinal Products for Human Use (CHMP) is based on two main studies:

• One is a Phase III trial comparing treatment with palbociclib and letrozole, an aromatase inhibitor, with letrozole treatment alone. 444 patients who received palbociclib in this testing lived on average 24.8 months without their disease getting worse, compared to 14.5 months in the group of 222 patients that received letrozole alone.
• The other study is a Phase III trial which compared treatment of fulvestrant together with palbociclib to treatment with only fulvestrant. 521 women were registered in this trial, despite of their menopausal condition. Initial results showed that 347 patients who received palbociclib had an average of 11.2 months without their disease getting worse compared to 4.6 months for 174 patients who only received fulvestrant.

The major side effects related with myelosuppression, a state in which the patient’s bone marrow produces less blood cells than normal. Other side effects included:

• Infections
• Fatigue
• Nausea
• Vomiting
• Inflammation of stomatitis
• Diarrhea
• Hair loss

The judgment approved by the CHMP at its September 2016 meeting is a mediator step on Ibrance’s trail to patient access. The CHMP opinion will now be sent to the European Commission for the acceptance and implementation of a decision on an EU-wide marketing authorization.

Date: 16/09/2016

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/09/news_detail_002604.jsp&mid=WC0b01ac058004d5c1

According to a meeting between the European Medicines Agency (EMA), the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) and the United States’ Food and Drug Administration (FDA) hosted by EMA on 1-2 September 2016, configuration of data requirements by regulators worldwide can put in to motivate the development of new antibiotics to fight antimicrobial resistance globally and protect worldwide public health.  Conversely, regulatory activities are only one factor of the inclusive and versatile response needed to support and step up development of new antibacterial medicines that meet patient needs.

The conclusions of the meeting will be presented at the G7 Health Ministers’ meeting, organized by the government of Japan in Kobe on 11-12 September 2016.

The three agencies also agreed in the meeting that some flexibility should be applied to the requirements for clinical development programs for antibacterial agents where treatment options for patients are partial due to antimicrobial resistance.

The participants discussed approaches to clinical trial design, including choice of endpoints, as well as post-authorization monitoring activities for antibacterial medicines.

They also recognized areas of closer alliance and harmonization of efforts to support the development of safe and helpful antibacterial treatments.

Date: 07/09/2016

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/09/news_detail_002596.jsp&mid=WC0b01ac058004d5c1

The European Medicines Agency’s (EMA) Committee for Advanced Therapies (CAT) is arranging a workshop on 15 and 16 November 2016 to talk about scientific and regulatory challenges of immunotherapy medicines based on genetically modified T-cells (white blood cells that normally fight off viruses and bacteria).

Immunotherapy is a type of cancer treatment that assembles the body’s own resistance mechanism to fight cancer. Immunotherapy medicines have considerably changed the therapeutic landscape, mainly for the treatment of patients with certain cancers, such as lung cancer or melanoma.

T-cell based immunotherapy is a modern approach where T cells from a patient’s blood are genetically engineered in a laboratory in order to allow them to recognize cancer cells through definite receptor proteins. In the body of a patient, the modified T-cells can then spot and destroy cancer cells.

T-cell immunotherapy medicines are being developed and are now being tested in clinical trials in a variety of cancers. However, there are still many scientific and regulatory challenges to be prevail over before these modern products can be brought to the market for the assistance of patients.

Therefore the open workshop aspires to smooth the progress of dialogue between the CAT and medicine developers from industry and academia on:

• present scientific growth;
• regulatory requirements for product manufacture and testing;
• Non-clinical studies and clinical expansion.

Date: 16/08/2016

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/08/news_detail_002591.jsp&mid=WC0b01ac058004d5c1

The European Medicines Agency (EMA) has implemented a new section to its guidelines on good pharmacovigilance practices (EU-GVP), named “Product- or population-specific considerations II: Biological medicinal products”. Good pharmacovigilance practices are a set of procedures intended to make sure the strength of the system of safety monitoring. The new chapter provides supervision on how to better check and control the safety of biological medicines to optimize the secure and efficient use of these products in Europe.

Biological medicines hold one or more active substances made by or resulting from a biological source, such as blood or plasma. Some of them may be already present in the human body and examples include proteins like insulin and growth hormone. The dynamic substances of biological medicines are larger and more composite than those of non-biological medicines. Only living organisms are able to reproduce such complication. Their density as well as the way they are produced may result in a degree of inconsistency in molecules of the same active substance, particularly in different batches of the medicine.

Therefore the direction seem to support those accountable for monitoring these medicines by:

• highlighting detailed issues and challenges for the pharmacovigilance of biological medicines, e.g. in relation to inconsistency of the active gist or tractability of products;
• providing proposals on how to deal with these specificities and challenges;
• Delineation the roles and responsibilities of the various actors.

The GVP guidance comes into force on 16 August 2016.

Date: 15/08/2016

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/08/news_detail_002590.jsp&mid=WC0b01ac058004d5c1

Publication of evaluation reports in Europe and Australia makes information on medicines simply available.

Today in a joint article published by Drug Discovery, the European Medicines Agency (EMA) and the Therapeutic Goods Administration (TGA) in Australia depict their constructive experiences with the publication of evaluation reports for medicines – known as European public assessment reports (EPARs) in Europe and Australian Public Assessment Reports (AusPARs) in Australia.

According to the authors, growing web traffic depicts the regulators’ success in assisting access to medicines’ information and the validations for medicines’ approval.

By focusing on authenticate, evidence-based, and up-to-date information, EPARs and AusPARs throw in to the propagation of consistent facts on medicines and how they are appraised and guarantee transparency. The reports build on close and reliable interface with a wide range of stakeholders in order to reflect the perceptions of different audiences.

Scientific development and stakeholders’ desire for greater information need a permanent development in the communication of information on medicines. Both regulators widely collect feedback on the value of the published information to evaluate continuously how to best communicate high-quality information on medicines to users and researchers.

Dated: 05/08/2016

http://www.ema.europa.eu/ema/index.jspcurl=pages/news_and_events/news/2016/08/news_detail_002587.jsp&mid=WC0b01ac058004d5c1

The European Medicines Agency (EMA) has commenced a public discussion on revised regulation on the development of new medicines to treat tuberculosis (TB). The regulation is an addition to EMA’s guideline on the assessment of medicines to treat bacterial infections.

TB is caused by a bacterium called Mycobacterium tuberculosis. In Europe, about 340,000 new TB cases and 33,000 deaths were reported in 2014, frequently from eastern and central European countries. While TB is slowly dilapidated worldwide, the trouble of the disease is still very high with just about 1.5 million deaths per year. Moreover, multidrug-resistant tuberculosis (MDR-TB) still poses a serious public health challenge. It often affects people from the most vulnerable communities, including migrant workers, refugees, displaced persons, prisoners or drug users.

Today’s TB treatments cannot efficiently fight the disease because they are long, intricate, and usually show abridged effectiveness against MDR-TB, striking a heavy burden on patients, families and healthcare systems. New TB medicines and regimens that are simpler to administer, are of shorter duration, and can conquer drug resistance are without delay needed.

In modern years, there has been a shift towards rising completely new regimens to treat TB, rather than focusing on single medicines. The revised guidance takes into account this expansion.

The direction also illuminates the European Union’s regulatory chucks with regard to data that should be produced to hold up the endorsement of new medicines and gives direction on the following topics:

• assessment of the effectiveness of entity new medicines and regimens in light of newly standard medicines;
• assessment of new regimens including at least one new medicine;
• Functioning of biomarkers to calculate the effectiveness of the medicine during clinical development.

EMA will arrange a workshop in November 2016 to confer stakeholders’ comments on the revised regulation.

Stakeholders can send their comments to the Agency until 31 January 2017.

Dated: 01/08/2016

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/08/news_detail_002583.jsp&mid=WC0b01ac058004d5c1

The MRHA has permitted Sanofi Pasteur MSD UK’s quadrivalent influenza vaccine, which includes two A strains (A/H1N1 and A/H3N2) and two B strains (B/Victoria and B/Yamagata) of the virus.

The mainstream of seasonal influenza vaccines in the UK are at this time trivalent, means they help out to protect aligned with three strains of the influenza vaccine; two a strains and a single B strain. However, in the UK’s 2015-2016 flu season 94 percent of influenza B cases belonged to the B/Victoria pedigree. This extraction was only incorporated in quadrivalent formulations and not incorporated in the trivalent vaccines suggested by the World Health Organization.

According to Sanofi Pasteur MSD, “Inclusion of the second B strain in quadrivalent influenza vaccines can help to provide broader protection by minimizing the impact of a mismatch of vaccine and disease-causing strains. It is predicted that quadrivalent vaccines will become a new standard in the preclusion of influenza and is devoted to work with healthcare professionals to facilitate and protect adequate populations.”

The company added that if quadrivalent vaccines were used in the UK over the past decade, it has been anticipated that an additional quarter of a million influenza cases could have been evaded, including 5,940 influenza-related hospitalizations and 3,955 influenza-related deaths.

Date: 26^th July’ 2016

http://www.pharmatimes.com/news/sanofi_pasteur_msds_four-strain_flu_vaccine_approved_1081255

The Cancer Research UK-funded study, published in Nature Communications, observe the outcome of the drug atovaquone on tumors with low oxygen levels in mice to notice that it would be helpful to treat cancer. According to research the drug decreases the speed at which cancer cells use oxygen by targeting the mitochondria which is the main powerhouses of the cell that make energy, a method that utilizes oxygen.

As radiotherapy works by damaging the DNA in cells, and a good supply of oxygen decreases the capability of cancer cells to fix broken DNA, tumors with low oxygen levels are more complicated to treat effectively with radiotherapy. By slow down the use of oxygen, atovaquone therefore reverses the low-oxygen levels in almost all of the tumors. The fully-oxygenated tumors are more effortlessly destroyed by radiotherapy.

The drug was revealed to be useful in a extensive range of cancers, including lung, bowel, brain, head and neck cancer.

“This is a stimulating result. We have now started a clinical trial in Oxford to see if we can prove the same results in cancer patients. We expect that this accessible low cost drug will mean that resistant tumors can be re-sensitized to radiotherapy. And we’re using a drug that we already know is safe,” said Lead author, Professor Gillies McKenna, at the Cancer Research UK/Medical Research Council Institute for Radiation Oncology in Oxford.

According to Dr Emma Smith, Cancer Research UK’s science information manager, “The types of cancer that tend to have oxygen dispossessed regions are often more difficult to treat – such as lung, bowel, brain and head and neck cancer. Looking at the cancer-fighting properties of existing medicines is a very important area of research where medical charities can make a big blow and is a main concern for Cancer Research UK. Clinical trials will tell us whether this drug could help recover treatment options for patients with these types of tumor.”

Date: 25^th July 2016

http://www.pharmatimes.com/news/anti-malaria_drug_could_help_fight_cancer_1080660