Pharming Group Receives Accelerated Assessment in Europe for leniolisib for the Treatment of Rare Immunodeficiency, APDS

 

 

Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has granted an accelerated assessment for the Marketing Authorization Application (MAA) for leniolisib. Leniolisib has been studied for the treatment of activated PI3K delta syndrome (APDS), a rare primary immunodeficiency, in adults and adolescents age 12 or older in the European Economic Area (EEA). Pharming is on track and plans to submit its MAA for leniolisib to the EMA in October 2022. 

Accelerated assessment reduces the timeframe for the CHMP to review an MAA from 210 days to 150 days. The EMA will grant, upon request, accelerated assessment of an MAA if they decide the product is of major interest for public health and therapeutic innovation.

The clinical development for leniolisib includes positive data from a Phase II/III study of the product, which met both its co-primary endpoints in the target patient population of evaluated reduction in lymph node size and correction of immunodeficiency. The primary efficacy results demonstrated clinical efficacy of leniolisib over placebo with a statistically significant reduction from baseline in the log10 transformed sum of product of diameters (SPD) in the index lymphadenopathy lesions (p=0.0012) and normalization of immune dysfunction, as evidenced by increased proportion of naïve B cells from baseline (p<0.0001). The shrinking of lymphadenopathy lesions and increased proportion of naïve B cells are important in patients as they indicate a reduction in APDS disease markers. 

In the study, leniolisib was generally well-tolerated, with the majority of reported adverse events in both treatment groups classified as mild. There were no adverse events that led to discontinuation of study treatment, there were no deaths, and the incidence of serious adverse events (SAEs) was lower in the leniolisib group than the placebo group. None of the SAEs were suspected to be related to study treatment.

Anurag Relan, Chief Medical Officer of Pharming, commented:

“The acceptance of an accelerated regulatory review for leniolisib underlines the high unmet need for patients with APDS, with the product potentially being the first approved treatment for this rare disease. This is an important milestone for the APDS community and for Pharming and is built on the successful Phase II/III data, which we first reported in February 2022. We remain focused on progressing leniolisib through the regulatory review process, with our MAA on track for submission in October of this year, as we seek to make this important new product available to immunologists, hematologists, and their patients in Europe.”

Encouraging developments in the area of national cancer screening programmes

The implementation report on the 2003 Council Recommendation on cancer screening, published today, reveals a steep rise in national screening programmes for breast, cervical and colorectal cancer.

Vytenis Andriukaitis, Commissioner for Health and Food Safety, said "Breast, cervical and colorectal cancer claim the lives of around a quarter of a million European citizens each year. Early detection - as well as cancer prevention - saves lives, and so this is where we must continue focusing our efforts. The Council's recommendation and the Commission's quality assurance guidelines have led to most EU countries putting in place organised, high quality screening programmes. This is a major step forward for citizens' health."

Today, 25 EU countries have population-based programmes for breast cancer, 22 for cervical cancer and 20 for colorectal cancer screening, as compared with 18, 17 and 12, respectively, since the first report was published 10 years ago.

Nearly 25 million women aged 50-69 years were invited to mammography screening in the last reporting year, and 110 million men and women aged 50-74 years could potentially undergo screening for colorectal cancer.

In addition to highlighting the status and volume of screening programmes in EU countries, today's report includes a set of essential indicators to document the quality of screening. Read the full report.

For more information:

http://ec.europa.eu/health/major_chronic_diseases/diseases/cancer_en#fragment4

https://www.iarc.fr/

 

Effective cancer control saves lives, money and time – quality in the core

 

A new Guide published online today gives recommendations on how to boost cancer control in Europe. The Guide is the result of a three-year effort by top experts in 25 countries and 126 partner organisations. They have been working together in an EU co-funded joint action, known as Cancon (officially titled European Guide on Quality Improvement in Comprehensive Cancer Control Guide).

- The quality improvement of cancer care is at the heart of the Cancon Guide. It will be reached by focusing on the patient’s experience, care and health services organization, delivery and accessibility, says Tit Albreht, the coordinator of Cancon joint action.

The Guide’s authors stress that besides saving money and time, effective cancer control increases quality of life. Currently some 2.6 million people in Europe are diagnosed with cancer annually. But more and more cancer patients are overcoming the disease. Therefore greater attention needs to be given to access to care, early diagnosis, rehabilitation, and survivorship.

- The good practice recommendations in the Guide strive to improve the national situations. The Guide is meant for decision-makers and cancer care professionals in Europe, explains Albreht.

The Guide includes a detailed survivorship care plan and evidence-based recommendations on cancer screening programmes for the member states of the European Union. This is the main outcome of the joint action.

The Guide will be officially launched at the Cancon Final conference, 14-15 February in Malta. The recommendations will be presented and discussed at the conference. This high-level conference includes a panel of European Health Ministers, and discussion on challenges in cancer policy at EU level.

- The implementation of these recommendations is the next step. We do not want the Guide to be just another book on the shelf, says Albreht.

**Cancon is a joint action initiative, co-funded by participating institutes, organisations, universities and health care units, and the European Union. 

First patient enrolled in the INNODIA European sample collection effort

Just one year after the Kick-off of INNODIA the IMI-supported European consortium has accomplished its first major milestone. INNODIA aims to improve the understanding of type 1 diabetes and is paving the way to novel therapeutic options to prevent and cure this disease. INNODIA will welcome the first patient to be included into the INNODIA European sample collection system on Worldiabetesday.

INNODIA will collect throughout Europe blood samples and data from newly diagnosed patients with type 1 diabetes and first degree relatives of people with type 1 diabetes and study the evolution of the disease in these individuals. ‘This way we will be able to better understand the relationship between changes in β-cell function, immune profiles, genetic and environmental factors and their role in the onset of the disease’ says Prof Chantal Mathieu, from the University of Leuven, Belgium and coordinator of INNODIA.

INNODIA has set up a network in Europe to collect samples, but also to perform coordinated clinical intervention studies of which the first interventions will start soon.

‘These are exciting times’ says Prof David Dunger, from the University of Cambridge, UK and coordinator of INNODIA’s clinical sample collection network. ‘In close collaboration with patients, we have developed protocols for follow up, information brochures, informed consents and materials necessary for this collection and will be creating a living biobank’. INNODIA joins forces between academic and industry researchers, but also involves patients and their families. ‘Listening to the patient’s voice and following their advice was crucial in setting up this effort’ concludes Dr. Olivier Arnaud from the JDRF International (formerly, The Juvenile Diabetes Research Foundation) and coordinator of the INNODIA Patient Advisory Committee.

Watch the INNODIA video and Kyle’s story reflecting on type1 diabetes and his role in INNODIA (www.innodia.eu )

Within the coming months the sample collection initiative will be expanded in more INNODIA centers across Europe opening the possibility for patients throughout Europe to participate. Check out the INNODIA website for upcoming news.

Thousands of melanoma patients in Europe have no access to new life saving drugs

  Over 5000 patients with metastatic melanoma in Europe are denied access to new, life saving drugs every year, according to a survey presented at the ESMO 2016 Congress in Copenhagen¹.

Metastatic melanoma is an aggressive and deadly skin cancer. With innovative targeted therapy and immunotherapy, patients can survive for many years. Unfortunately new therapies are expensive so, according to a survey conducted by Dr Lidija Kandolf-Sekulovic, over 5000 patients with metastatic melanoma in Europe have no access to these drugs.

“Before 2011 there were no effective treatment options for metastatic melanoma patients, but that has changed tremendously in the last 5 years. We now have medicines which can prolong overall survival of these patients to more than 18 months and, in some patients, durable responses lasting up to 10 years have been reported. However, access to these medicines is limited and patients and physicians are facing increasing difficulties to obtain them. This is especially the case for Eastern and South Eastern European countries, where a majority of patients are still treated with palliative chemotherapy that does not prolong overall survival,” said Kandolf-Sekulovic.

The survey showed that in Western Europe 70% of patients were treated with innovative medicines, while in Eastern Europe less than 10% of patients had access to the latest treatment recommended by current European Guidelines (ESMO, EORTC/EADO) (2).

The study found that the BRAFi+MEKi combination (one of the first-line treatments besides immunotherapy for BRAF mutated metastatic melanoma) was registered in 75% of Western European countries and fully reimbursed in 58%. In Eastern Europe, the treatment was registered in 42% of countries and only reimbursed in 18%, with time consuming administrative work needed to obtain the medicines in all cases.

The survey estimated that around 19.250 metastatic melanoma patients are treated every year in Europe and nearly 7.450 (39.7%) in Eastern and South-Eastern Europe. Of these patients, 5.128 (69%) do not have the access to first-line therapy according to European guidelines. Overall, it can be estimated that in Europe 5.228/19.250 (27%), i.e. almost one third of all metastatic melanoma patients, do not have access to innovative medicines.

In Europe, about 1 in every 100 people will develop melanoma at some point in their life, but important variations exist from one country to another. This number is increasing in almost all European countries. Melanoma is slightly more frequent in females than in males and more frequent in Switzerland, the Netherlands and the Scandinavian countries (Norway, Sweden and Denmark), where about 20 out of 100,000 people are diagnosed each year. From 1999-2012 there has been a 78% recorded increase in Germany. Similar increases were recorded also in the United States, Australia, Norway and Denmark, as well as countries in South-Eastern Europe.

Kandolf-Sekulovic explained: “Our study raises ethical questions on the inequalities that affect survival based on the country of residence in Europe. It is not new that disparities in healthcare can lead to disparities in overall survival of patients, but these disparities are becoming even sharper for patients with chemotherapy resistant metastatic melanoma in whom durable responses lasting for years can be seen in up to 20% of patients if treated with innovative medicines. In European healthcare systems that declare universal access to healthcare, these inequalities must be overcome.”

Dr Alexander Eniu, Chair of the ESMO Global Policy Committee, said: “This study confirms what ESMO has highlighted in the past: access to the best treatment according to evidence based clinical guidelines such as ESMO’s, is not equal across Europe. ESMO advocates for equal access to treatment and care, which is the fundamental right of any patient.  Despite the encouraging rate of new medicine development, there are still unacceptable inequalities in the availability and accessibility of new and effective cancer medications across Europe.”

 “The present study focuses on melanoma but the ESMO-led European Consortium Study on the availability and accessibility of anti-neoplastic medicines across Europe (3) found that the same was true for other types of cancer, especially rare cancers, in countries with lower economic levels.  It is important to continue to provide health authorities with data, and to carry on calling attention to the difficulties patients with incurable diseases are facing, in the hope that equal access will soon be a reality, at least in Europe,” said Eniu.

“This every day situation which is source of a large frustration for metastatic melanoma patients, their families and physicians, needs to be adressed urgently by all stakeholders. We need harmonisation of reimbursement procedures throughout Europe, adjusted programmes for early access to innovative medicines in countries with delayed reimbursement and sustainable pricing for these life saving drugs,” concluded Kandolf-Sekulovic.

Study finds kidney transplant donation rates vary widely across Europe

A new Europe-wide survey shows significant country-to-country differences in rates of kidney transplant donors. The survey shows for example within the EU, there is a x5 variation in the number of kidney donors per country (per head of population). This variation is probably due to different legal and social standards across Europe.

Kidneys can fail for a variety of reasons, the most common being diabetes, high blood pressure, drug overdose, and physical injury. Kidney dialysis is often used as a treatment, but the best long-term solution is transplant, usually from a recently-deceased donor. Rates of Chronic Kidney Disease vary considerably across Europe, from 3% to 17% of the population, and are increasing. * Demand for kidneys almost always exceeds possible supply, and each country manages transplants differently. Now a new survey, being presented at the European Association of Urology congress in Munich, has shown significant differences in the number of donor kidneys available in each country.

Dr. Víctor Díez Nicolás (associated member, European Society for Transplantation in Urology, ESTU) will present the results of this ESTU-led survey on kidney donation rates and kidney transplantation rates across Europe for 2014. Comparing data from a range of registers, they found wide country to country variation. For example:

Russian Federation        3.3          All figures are deceased donors per million population

Greece                                4.2

Germany                            10.4

Switzerland                       14.3

Poland                                 15.5

The Netherlands             16.8

UK                                         20.6

Italy                                      22.7

France                                 25.3

Portugal                              27.3

Croatia                                 35.1

Spain                                    35.7                                                                      All figures are 2014 figures

Note: All- country data are in the abstract

Dr Díez Nicolás said:

“The circumstances in country are very different, but basically it depends on two factors, social sensitivity, and legislation. Most organs for transplantation are come from brain dead donors.  But each country manages organ availability differently. In Spain for example, each citizen is a potential donor unless they opt-out of the transplant scheme, whereas in Germany there is an ‘opt-in’ scheme. Some countries also allow donation from living donors, or from persons whose hearts have stopped. The number of organs from both these sources has been increasing”.

As the technology becomes more mainstream, and rates of kidney failure are increasing, the demand for organs has increased quite significantly, and there is a general need to obtain more organs. At the moment, whether you can find a donor organ largely depends on where you live. If countries want to increase transplant rates, and so increase survival from kidney failure, they might consider changing the way they source donor organs”.

Professor Arnaldo José Figueiredo (Coimbra, Portugal), chairman of the ESTU Said:

“Transplantation remains the most effective way of replacing kidney function. End stage renal failure incidence is increasing steadily in all European countries and, notwithstanding the fact that efforts should primarily be put on prevention, transplantation activity also needs be promoted, as demand clearly exceeds supply. There are significant discrepancies in transplant activity among European countries, and the ones with lower rates of transplant per capita should look at the examples of the leaders. At the same time, there are important differences where the transplanted organs come from, such as brain dead, non-heart beating and living donors, meaning there is scope for improvement in each program”.

There was no external funding for this research.