Early Access Program With Arimoclomol in US Patients With NPC

Open to people ages 2 years and up

• The patient has a confirmed diagnosis of NPC (NPC1 or NPC2)* and at least one neurological symptom.
• The patient is two years of age or above.
• The patient is a permanent resident of US.
• If taking miglustat (Zavesca®), the patient must have been on the target dose for the past six weeks.
• If the patient is a sexually active female of child-bearing potential (post-menarche), it is agreed to use highly effective contraception during the EAP and until three weeks after the last dose of arimoclomol.
• Confirmed negative urine pregnancy test for sexually active female of child-bearing potential (post-menarche).
• All sexually active male patients with female partners of child-bearing potential (postmenarche) agree to use a condom in addition to the birth control used by their partners during treatment and until three weeks after the last dose of arimoclomol.
• If the patient has a history of seizures, the condition must be adequately controlled, i.e., the pattern of seizure activity must be stable, and the patient must be on a stable dose and regimen of antiepileptic medication during one month prior to screening.
• Patient or parent/guardian must provide written informed consent to participate in EAP.
  • In line with Patterson et al. 2017

• Severe liver insufficiency.
• Renal insufficiency.
• The patient has a known or suspected allergy or intolerance to arimoclomol or its constituents.
• The patient is pregnant, planning to become pregnant (while on the EAP program) or is currently breastfeeding.
• The patient will undergo treatment with another investigational drug*, whilst participating in the program or in the 4 weeks prior to commencing treatment with arimoclomol.
• The patient is either eligible and able to participate in or is currently participating in an active interventional clinical trial within the indication.
• The patient, in the opinion of the clinician, is unable to comply with the treatment or has a medical condition that would potentially increase the risk to the patient by participation.
• The patient has a medical condition which hinders the clinician's assessment of arimoclomol safety and efficacy (e.g. certain epileptic conditions or severe cataplexy).
  • Including unlicensed product provided under an Early Access Program or equivalent compassionate use programs