My Story with NITYR® (nitisinone) Tablets

Five-year-old Ethan has Tyrosinemia Type 1 (HT-1). Like any family that has a child with this ultra rare condition, the family have found Ethan’s diet and medication regime very demanding.

"My son was really excited to be able to swallow the medication."

Ethan started on Orfadin®* (nitisinone) capsules as a baby, which his parents had to mix with water to create a suspension to syringe into his mouth. The medication, nitisinone, sometimes known as NTBC, needed to be kept in a refrigerator, which the busy family found restricting, as it meant carrying ice packs around when travelling even short distances.

Convenient to Use

When Ethan’s parents, Carrie and Mike, heard that NITYR was bioequivalent to Ethan’s current medication Orfadin®*, but had some advantages that may be more convenient to them, they wanted to know more.1

Ethan’s dietitian encouraged the family to look into NITYR, as she felt he was ready to swallow a tablet. The small size of the NITYR tablet would be perfect for this.2

"What I like about NITYR is it’s a really small2 tablet,” says Carrie. “It comes in the size of baby aspirin, and it can be taken with or without food any time of day. It doesn’t need to be refrigerated."

Swallowing Whole Tablets

Ethan was soon able to swallow the small NITYR tablets without any trouble at all. Like Carrie and Mike, parents of young children should always consult a healthcare provider before getting their child to attempt to swallow a tablet.

"My son was really excited to be able to swallow the medication,” says Carrie. “He shows everyone that comes over how big he is, when he gets to take his medication with water. He asks for a crowd! He’s really proud of himself. He feels like a big boy, really independent."

Carrie and Mike have a message for other families who are considering moving to NITYR.

"For a family that’s unsure about switching to NITYR, I think I simply would say just give it a try. There is nothing to lose, you have everything to gain,” says Mike. “I think it’s important for caregivers to be open to innovation, because we are such a small community that we don’t always get the attention as some other conditions."

"I would tell a new family not to be afraid and to give it a try," adds Carrie. "It was a really positive experience for my son."

*Orfadin is a registered trademark, licensed by Swedish Orphan Biovitrum AB (publ) and is marketed by Sobi, Inc.

At the time of filming, Ethan was taking NITYR. Ethan, Carrie and Mike were compensated for their time.

References

1. Data on file: REF-00047
2. Data on file: REF-00041