Arcalyst

Question 1

What are the risks of ARCALYST during pregnancy?

Accepted Answer

There have been rare pregnancy outcomes reported postmarketing and from clinical trials, with very limited use of ARCALYST in pregnant women, so there is insufficient information to evaluate for a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. In an animal reproduction study, subcutaneous administration of rilonacept to pregnant monkeys during the period of organogenesis was associated with a dose-related increase in exposure but no treatment-related effects on fetal survival or development of malformations with doses up to 11 times the maximum recommended human dose (MRHD). Increased incidences of lumbar ribs, a skeletal variation, were observed in fetuses at doses approximately 2 times the MRHD and higher, and there were findings of multiple fusion and absence of the ribs and thoracic vertebral bodies and arches in one fetus of the only pregnant monkey with exposure to rilonacept during the later period of gestation associated with a dose approximately 6 times the MRHD. The relationship of these findings in a single fetus to drug treatment was unclear, as these findings were not evident in fetuses from pregnant monkeys that had higher exposures to rilonacept during the period of organogenesis associated with a dose approximately 11 times the MRHD. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. The estimated background risk of major birth defects and miscarriage for the indicated populations is unknown. In the U.S. general population, the estimated background risks of major birth defects and miscarriage in clinically recognized pregnancies is 2%–4% and of miscarriage is 15%–20%, respectively.

Question 2

What should I consider if I am breastfeeding while using ARCALYST?

Accepted Answer

There is no information on the presence of rilonacept in either human or animal milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for ARCALYST and any potential adverse effects on the breastfed child from ARCALYST or from the underlying maternal condition.

Question 3

Is ARCALYST safe and effective for pediatric patients with CAPS and RP below the age of 12 years?

Accepted Answer

No, safety and effectiveness in pediatric patients with CAPS and RP below the age of 12 years have not been established for ARCALYST.

Question 4

What subcutaneous dose of ARCALYST was used to treat pediatric patients with CAPS in the open-label extension phase?

Accepted Answer

Six pediatric patients with CAPS between the ages of 12 and 16 were treated with ARCALYST at a weekly subcutaneous dose of 2.2 mg/kg (up to a maximum of 160 mg) for 24 weeks during the open-label extension phase.

Question 5

What were the results of treating pediatric patients with ARCALYST?

Accepted Answer

Pediatric patients with CAPS treated with ARCALYST showed improvement from baseline in their symptom scores and in objective markers of inflammation (e.g., Serum Amyloid A and C-Reactive Protein). The adverse reactions included injection-site reactions and upper respiratory symptoms as were commonly seen in adult patients.

Question 6

What is the recommended monitoring for pediatric patients treated with ARCALYST?

Accepted Answer

Pediatric patients treated with ARCALYST should undergo appropriate monitoring for growth and development.

Question 7

Is ARCALYST safe and effective for pediatric patients with DIRA weighing less than 10 kg?

Accepted Answer

No, safety and effectiveness of ARCALYST have not been established in pediatric patients weighing less than 10 kg for maintenance of remission of DIRA.

Question 8

What is known about the safety and effectiveness of ARCALYST in geriatric patients?

Accepted Answer

In the placebo-controlled clinical studies, 78 patients randomized to treatment with ARCALYST were  65 years of age, and 6 were  75 years of age. In the CAPS clinical trial, efficacy, safety, and tolerability were generally similar in elderly patients as compared to younger adults; however, only ten patients  65 years old participated in the trial. In an open-label extension study of CAPS, a 71-year-old woman developed bacterial meningitis and died. Age did not appear to have a significant effect on steady-state trough concentrations in the clinical study.

Question 9

Has the pharmacokinetics of rilonacept administered subcutaneously been evaluated in patients with renal impairment?

Accepted Answer

No, no studies have been conducted to evaluate the pharmacokinetics of rilonacept administered subcutaneously in patients with renal impairment.

Question 10

Has the pharmacokinetics of rilonacept administered subcutaneously been evaluated in patients with hepatic impairment?

Accepted Answer

No, no studies have been conducted to evaluate the pharmacokinetics of rilonacept administered subcutaneously in patients with hepatic impairment.