Daily Brief: Jazz Pharma and Ultragenyx approvals, Autonomous AI in retinopathy

Jazz Pharma gains EU approval for niche AML treatment

Jazz Pharmaceuticals has gained EU approval for Vyxeos, its treatment for adults with newly diagnosed, therapy-related acute myeloid leukaemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC).

The new approval is part of new a wave of drugs for AML, which has been lacking new treatment options until now.

Therapy related AML accounts for around 10% of all AMLs, and are caused by a patient's exposure to chemotherapy and/or radiation for a primary malignancy or autoimmune disease.

Vyxeos is a liposomal formulation combination of existing chemotherapies daunorubicin and cytarabine.

“Vyxeos is the first chemotherapy to demonstrate an overall survival advantage versus the standard of care in a phase III study of older adult patients with newly diagnosed therapy- related AML or AML with myelodysplasia-related changes,” said Daniel Swisher, president and chief operating officer at Jazz Pharmaceuticals.

The company says it will now pursue rolling launches of Vyxeos across the EU on a country-by-country basis as pricing and reimbursement decisions are made.

The pivotal phase III study evaluated the efficacy and safety of Vyxeos compared to 7+3 chemotherapy in 309 patients aged between 60-75 with newly diagnosed t-AML or AML-MRC, a rapidly progressing and life-threatening blood cancer.

The median overall survival for the Vyxeos treatment group was 9.6 months compared with 5.9 months for the 7+3 treatment group.

Vyxeos was also associated with a higher remission rate than 7+3, with a complete response rate of 37% versus 26%; p=0.036.

In addition, the overall rate of haematopoietic stem cell transplant (HSCT) was 34% in the Vyxeos arm and 25% in the 7+3 arm. The reported adverse reactions with Vyxeos were generally consistent with the known safety profile of cytarabine and daunorubicin therapy.

Ultragenyx gains EU approval for rare disease drug Mepsevii

Ultragenyx has gained EU approval for Mepsevii in the EU, the first treatment approved in Europe for Mucopolysaccharidosis VII (MPS VII).

The disease was first described in 1973 by William Sly, chairman emeritus at St. Louis University's Department of Biochemistry in 1973, and he has worked for decades to bring an enzyme replacement therapy to patients.

Mepsevii (vestronidase alfa) was approved by the FDA in November 2017 and has been priced at around $375,000 per patient per year after discounts in the US.

“Dr William Sly’s science for this treatment has been around for over 25 years and we are honoured to be able to develop and finally make this medicine available to MPS VII patients and families in Europe, people who did not know whether a therapy would ever be available to them,” said Emil Kakkis, CEO and President of Ultragenyx.

“This would not have been possible without the commitment and dedication of the patients, their families, and the physicians who participated in our clinical programme, and I would like to thank them.”

Another variant of the disease, MPS IVa already has a treatment available – Biomarin’s Vimizim.

First ever autonomous AI success in diabetes scan

An artificial intelligence (AI) system that scans patients at risk of diabetic retinopathy without any human assistance has performed well in an early trial.

The company behind the technology is IDx and has published results from the study of its IDX-DR system in Nature Digital Medicine.

The FDA cleared the diagnostic for use in April this year, and the University of Iowa Health Care started using the system in regular clinical practice in June.

It says the pivotal trial results assessing the safety and efficacy of the system is the first ever study of a completely autonomous AI system in healthcare.

"This is formerly uncharted territory in healthcare, making it especially critical that we ensure the highest level of safety before introducing autonomous AI into patient care," said Michael Abràmoff, the study's principal investigator and the founder and president of IDx.

"That's why it was so important for us to develop an exceptionally rigorous study that was reviewed by independent physician-scientists. Now that the results have been published in Nature Digital Medicine, scientists, physicians and patients can all evaluate the scientific evidence for the safety and effectiveness of an autonomous AI like IDx-DR."

In the clinical study, IDx-DR achieved high diagnostic accuracy when compared to the most rigorous determination of diabetic retinopathy severity using advanced imaging techniques – wide-field stereo fundus imaging and optical coherence tomography (OCT), which were evaluated by the Wisconsin Fundus Photograph Reading Center.