Bluebird Bio announces launch of Lentiglobin BB305 gene therapy, branded as ZYNTEGLO, in Germany. This is the first time ZYNTEGLO is commercially available and is priced at 1.575 million euros (1.77 million dollars) per patient.
ZYNTEGLO received conditional marketing authorization by the European Commission in June 2019 as a one-time gene therapy designed to treat all but the most severe forms of transfusion-dependent beta-thalasssaemia in people over 12 years of age, for whom haematopoietic stem cell transplantation from a matched donor is not an option. The company has recruited the German manufacturer apceth Biopharma to produce ZYNTEGLO and is working with specialized institutions in Germany to establish qualified treatment centers. The University Hospital of Heidelberg is the first such center to administer the therapy. The company is negotiating with multiple statutory health insurance providers in Germany to establish a value-based payment agreement where the sum will be broken up into one 315,000 euros installment per year for five years. After the first installment is paid, subsequent installment payments will be made only if the therapy prevents the need for blood transfusions completely.
Source: Bluebird Bio press release here and here.


The Cooley’s Anemia Foundation (CAF) is accepting applications for its 2019-2020 "Patient Incentive Awards" and "Educational Incentive Awards for Children of Individuals with Thalassaemia". The aim of these awards is to support patients and their families to further their education and career goals towards more fulfilling, active lives.
Application Deadline: February 14, 2020
Eligibility: U.S. residents currently enrolled in a graduate, undergraduate, associate, certificate or vocational training programme.
Requirements: Patient incentive Award, Educational Incentive Award Program for Children of Individuals with Thalassemia

Oxbryta™ is an oral medication for the treatment of sickle cell disease (SCD) by directly inhibiting sickle haemoglobin polymerisation. As a condition for having received accelerated approval from the FDA, Oxbryta™ will be assessed in a post-approval confirmatory study, called HOPE-KIDS 2, which is set to launch by the end of the year. The trial will test Oxbryta™ tablets at a daily dose of 1500 mg, its approved dose, in pediatric patients (age 2 to 15) with SCD, and its primary goal will be to demonstrate that treatment with Oxbryta™ can reduce the risk of stroke in SCD children at 24 weeks of treatment. The trial will use transcranial doppler (TCD) to measure flow velocity. The trial will run at 50 sites across the U.S., Europe, and Africa. For more info see here.

La Jolla Pharmaceutical Company has announced plans to discontinue Study LJ401-BT01 due to mixed clinical results. LJPC-401 is a synthetic human hepcidin for the treatment of conditions characterized by iron overload. Study LJ401-BT01 (NCT03381833) is designed to evaluate the safety and efficacy of LJPC-401 as a treatment for iron overload in beta thalassemia patients who, despite chelation therapy, have cardiac iron levels above normal. A recent interim analysis involving half of the enrolled population suggested lack of efficacy, with patients on the treatment and control arms exhibiting similar changes in cardiac iron levels, the primary endpoint, as well as key secondary endpoints. For more information, read La Jolla News here.

Voxelotor, marketed under the brand name Oxbryta™ (Global Blood Therapeutics Inc., GBT), has received Accelerated Approval from the U.S. Food and Drug Administration (FDA) for the treatment of adults and pediatric patients aged 12 years and older with sickle cell disease (SCD). Oxbryta™ is a once-daily pill that works by directly inhibiting sickle haemoglobin (Hb) polymerization, the main cause of SCD. The drug’s approval came three months earlier than expected and is based on results from the randomized, global Phase 3 HOPE trial (NCT03036813). Trial findings were published in The New England Journal of Medicine, showing clinically meaningful and statistically significant improvements in Hb levels, accompanied by reductions in red blood cell destruction (hemolysis). Briefly, 51.1% of patients receiving Oxbryta™ for 24 weeks at 1500 mg daily, its now approved dose, achieved a greater than 1 g/dL increase in Hb compared with 6.5% receiving placebo (n=274, P<0.001). Common side effects for patients taking Oxbryta™ were headache, diarrhea, abdominal pain, nausea, fatigue, rash and pyrexia (fever). In spite FDA Fast Tract designation, further clinical trials are required to verify and describe the drug's clinical benefit. Oxbryta™ also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. For more information, read GBT press release here and FDA press release here