BioInItaly Newsletter

La vetrina del biotech italiano all’estero

N.5/ NEWSLETTER MENSILE 2017

12 giu, 2017

Newron Pharmaceuticals Announces Expansion of STARS Study to Include Patients Under 13 Years of Age  Recruitment for potentially pivotal study in patients suffering from Rett syndrome ongoing in the USA, Europe, Asia and Australia 
Newron Pharmaceuticals S.p.A. (“Newron”), a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central nervous system (CNS) and pain, announced that the Sarizotan Treatment of Apneas in Rett Syndrome (STARS) study has been expanded to include Rett syndrome patients as young as six years of age. An amendment to allow the inclusion of the patients under the age of 13 was approved by the FDA after Newron submitted results from juvenile toxicology studies. Newron is also submitting the amendment to Health Authorities and Institutional Review Boards in other countries. More info: http://www.newron.com/en

DiaSorin launches the new Liaison Androstenedione test for the evaluation of adrenal and gonadal functionality 
DiaSorin (FTSE Italy Mid Cap: DIA) is pleased to announce the launch of the new LIAISON test for the determination of Androstenedione in human serum and plasma, available worldwide with the exception of the U.S. The Androstenedione, produced by the adrenal glands and by the ovaries and the testicles, is a hormone responsible for the onset of sexual differentiation and for stimulating the development of male secondary physical characteristics, such as a deep voice and facial hair. Particularly, elevated Androstenedione levels can be associated to male physical characteristics (virilization) in young girls.
Additionally, elevated Androstenedione levels in blood are associated with hormone-related diseases such as polycystic ovarian syndrome (PCOS), congenital adrenal hyperplasia (CAH) and adrenal gland tumors. The new LIAISON® Androstenedione test expands the Company Fertility panel on CLIA technology and it is being recognized as the fastest assay available on the market. The European infertility market is estimated at 130 million euros1 with the Androstenedione accounting for about 2%2, equal to a volume exceeding 1.5 million tests per year. More info: http://www.diasorin.com/en/press-releases/diasorin-launches-new-liaison-androstenedione-test-evaluation-adrenal-and-gonadal

Sandoz proposed biosimilars adalimumab and infliximab accepted for regulatory review by the European Medicines Agency 
Sandoz, a Novartis division, and the pioneer and global leader in biosimilar medicines announced that the European Medicines Agency (EMA) has accepted for regulatory review their Marketing Authorization Applications for biosimilars to AbbVie's Humira® (adalimumab) and Janssen's Remicade® (infliximab) both of which are used to treat immunological diseases. Sandoz is seeking approval for biosimilar adalimumab and infliximab for use in all indications of their respective reference medicines. The comprehensive data packages included in the EMA submissions demonstrate biosimilarity of the proposed biosimilars to their respective reference medicines, with analytical, preclinical and clinical data matching across quality, efficacy and safety. More info: www.novartis.com/news/media-releases/sandoz-proposed-biosimilars-adalimumab-and-infliximab-accepted-regulatory-review

Dompé receives positive CHMP opinion in Europe for Oxervate® (cenegermin eye drops) for the treatment of adult patients with moderate or severe neurotrophic keratitis
The biopharmaceutical company Dompé has announced that the Committee for Human Medicinal Products (CHMP) of EMA (European Medicines Agency) has released a positive opinion, recommending the marketing authorization for Oxervate® (cenegermin eye drops), for the treatment of adult patients with moderate or severe neurotrophic keratitis. This is a rare and disabling eye condition that can lead to the loss of vision. As of today, no satisfactory treatment is available for this disease. Due to the risks related to this pathology and to the lack of viable therapeutic options, CHMP completed its review through an accelerated assessment. It began in November 2016 and ended with a positive opinion some days ago. More info: http://www.dompe.com/news-en/628_dompe-receives-positive-chmp-opinion-in-europe-for-oxervate.html

Novartis drug Tasigna receives EU approval for inclusion of Treatment-free Remission (TFR) data in product label  
Novartis announced that the European Commission (EC) has approved the inclusion of Treatment-free Remission (TFR) data in the Tasigna® (nilotinib) Summary of Product Characteristics (SmPC). TFR is the ability to maintain molecular response (MR) after stopping tyrosine kinase inhibitor (TKI) therapy in Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) patients in chronic phase (CP)[1]. This is an important milestone for the Ph+ CML community because Tasigna is now the first and only TKI to include information on TFR in the European Union (EU) label. The approval by the EC was based on efficacy and safety findings from the 48-week analyses of two open label trials, ENESTfreedom and ENESTop, which showed that more than 50% of Ph+ CML-CP patients who met the rigorous predefined response criteria of the trials were able to maintain TFR after stopping Tasigna in both in the first-line setting and after switching from Glivec® (imatinib)*[2],[3]. No new major safety findings were observed in these studies at the 48-week analyses in patients treated with Tasigna beyond those in the known safety profile of Tasigna[2],[3]. An important part of the ENESTfreedom and ENESTop trials was regular and frequent molecular monitoring of BCR-ABL levels with a well-validated assay able to measure BCR-ABL transcript levels down to MR4.5 (BCR-ABL1 International Scale [IS] <= 0.0032%). Frequent patient monitoring after discontinuation of Tasigna allows timely determination of loss of MR4.0 (BCR-ABL1 IS <= 0.01%) and major molecular response (MMR; BCR-ABL1 IS <= 0.1%) and need for treatment re-initiation[2],[3]. More info: www.novartis.com/news/media-releases/novartis-drug-tasigna-receives-eu-approval-inclusion-treatment-free-remission