CRYSVITA was effective in treating adults with TIO

Two clinical studies of adults with TIO showed that CRYSVITA may help by:

Increasing and maintaining phosphorus levels in the blood

Helping to heal soft or weak bone (osteomalacia)

How CRYSVITA was studied

The benefits and risks of treatment with CRYSVITA were looked at in a total of 27 adults with TIO

Study 6

Number of adults

14

on CRYSVITA

Ages

33-68

years old

Length of treatment

144

weeks

Study 7

Number of adults

13

on CRYSVITA

Ages

41-73

years old

Length of treatment

88

weeks

Phosphorus level results

CRYSVITA increased phosphorus levels in adults with TIO

In studies of CRYSVITA, adults with TIO had the level of phosphorus in their blood measured through week 144 (Study 6) and week 88 (Study 7).

Study 6

CRYSVITA helped to increase and maintain phosphorus levels in the blood within or near the normal range

This chart shows the increase in average phosphorus levels in the blood for people in Study 6 (14 adults, ages 33-68 years).

Phosphorus levels in the blood increased from an average of 1.60 mg/dL at baseline to 2.64 mg/dL averaged across the midpoint of dose intervals through week 24 and were sustained near or within the normal range through week 144

CRYSVITA

50%

7 out of 14 people had serum phosphorus levels in the normal range averaged across the midpoint of dose intervals through week 24

Study 7

Phosphorus levels in the blood increased from an average of 1.62 mg/dL at baseline to 2.63 mg/dL averaged across the midpoint of dose intervals through week 24 and were sustained through week 88

CRYSVITA

69%

9 out of 13 people had serum phosphorus levels in the normal range averaged across the midpoint of dose intervals through week 24

Osteomalacia results

CRYSVITA helped heal osteomalacia in adults with TIO

Under normal circumstances, minerals like phosphorus are continuously added to bones. This process, called mineralization, helps keep bones hard. People with TIO don’t have enough phosphorus for bones to properly mineralize, which causes some parts of the bone to become “soft” and more likely to break.

Study 6

CRYSVITA helped improve bone mineralization in people with TIO

CRYSVITA

34%

reduction in unmineralized or soft bone

In Study 6, a study of 14 adults with TIO aged 33-68 years, small samples of bone were taken and examined for signs of healing of osteomalacia. Bone samples were taken before patients started treatment with CRYSVITA and then again after the patients were treated with CRYSVITA every 4 weeks for 48 weeks.

Study 7 also showed improvement in bone mineralization with CRYSVITA

  • Study 7: 3 patients had bone samples taken both before and after treatment with CRYSVITA. In these patients, OV/BV was reduced by 34% after 48 weeks of CRYSVITA treatment

CRYSVITA reduced bone abnormalities caused by osteomalacia.

Bone scans taken during Study 6 showed that some people receiving CRYSVITA saw healing in their bone abnormalities at week 48 through week 144.