FDA approves first new sickle cell disease treatment in 20 years
The FDA has approved L-glutamine oral powder (Endari), a product of Emmaus Life Sciences Inc., for patients age 5 years and older with sickle cell disease (SCD) to reduce severe complications associated with the blood disorder. The approval is the first treatment for SCD in almost 20 years. Prior to its approval, only one other treatment existed for patients living with SCD.
The L-glutamine oral powder, an amino acid (which received orphan drug designation) reduces oxidative damage to red blood cells by improving the redox potential of nicotinamide adenine dinucleotide, a coenzyme that has been identified as the primary regulator of oxidation.
FDA approval was supported by efficacy data from a 48-week randomized, double-blind, placebo-controlled, multicenter phase III clinical trial evaluating the effects of L-glutamine oral powder compared with placebo on 230 adults and children with SCD. The results showed that the oral powder reduced the frequency of sickle cell crises by 25% and hospitalizations by 33%. Additional findings showed a decrease in cumulative hospital days by 41% and lower incidence of acute chest syndrome by more than 60%.
Common adverse reactions of L-glutamine oral powder include constipation, nausea, headache, abdominal pain, cough, pain in the extremities, back pain, and chest pain.
Novo Nordisk warns of possible cracks in insulin devices
Novo Nordisk warned patients with diabetes mellitus that the insulin cartridge holder used in a select number of NovoPen Echo lots may crack or break if exposed to certain chemicals, including some cleaning agents. The company is therefore issuing a global recall due to potential health risks. Using a device with a cracked/broken cartridge holder may result in the device delivering a reduced dose of insulin, which could potentially lead to hyperglycemia. The company has received numerous complaints of damaged cartridge holders and some reports of adverse events to date.
Affected batches were issued between August 1, 2016, and June 22, 2017, to distributors, sales representatives, and replacement programs for further distribution to pharmacies, healthcare providers, and patients nationwide. The company is notifying distributors, pharmacies, healthcare providers, and patients via mail and is arranging for product replacement.
Replacement cartridge holders will be provided for NovoPen Echo from the following batch numbers: EVG1221, EVG1226, FVG7149, FVG7458, FVG8134, FVG8135.
If patients have a NovoPen Echo device with a batch number that is not mentioned above, there is no reason for concern, and the pen will work as intended, the company states. Novo Nordisk has corrected the problem and has determined no other component of the pen is affected.
Abatacept approved for psoriatic arthritis
Bristol-Myers Squibb's abatacept (Orencia) received FDA approval for the treatment of active psoriatic arthritis (PsA) in adults. Abatacept is a selective T-cell costimulation modulator that is already approved for the treatment of rheumatoid arthritis (RA) and juvenile idiopathic arthritis.
The approval of abatacept for the PsA indication was based on results from two randomized, double-blind, placebo-controlled trials in which abatacept reduced disease activity in adult PsA patients with active musculoskeletal symptoms.
Abatacept is available in both I.V. infusion and subcutaneous injection formulations and should not be administered concomitantly with tumor necrosis factor inhibitors and is not recommended for use concomitantly with other biologic RA therapy, such as anakinra (Kineret).
The most serious adverse reactions reported in clinical studies in adult patients with RA were serious infections (3% abatacept versus 1.9% placebo) and malignancies (1.3% abatacept versus 1.1% placebo).
Headache, upper respiratory tract infection, nasopharyngitis, and nausea were the most commonly reported adverse reactions (10% or greater) in the adult RA clinical studies.