Early Access Program With Arimoclomol in US Patients With NPC

Inclusion Criteria:

- 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

Exclusion Criteria:

- 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