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Important Safety Information: Hypersensitivity reactions, including anaphylaxis have occurred. The most serious adverse reactions in patients treated with VPRIV (velaglucerase alfa for injection) were hypersensitivity reactions. Read more Important Safety Information

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VPRIV Efficacy by the Numbers

Efficacy of VPRIV in Treatment-Naïve Patients

In the overall population of treatment-naïve patients from Studies 032 and 039:

Mean Increase in Hemoglobin Concentration from baseline1,2

+23% at 12 months in Study 032 (n=12) P<0.0013,4

  • Mean hemoglobin concentration at baseline: 10.6 g/dL
  • Mean change from baseline to 12 months for patients receiving the 60 U/kg dose: 2.4 g/dL ± 0.3 (standard error)

+26% at 24 months in Study 044 (n=38)1,2*

  • Mean hemoglobin concentration at baseline: 11.04 g/dL
  • Mean change from baseline at 24 months: 2.75 g/dL; maintained stability vs baseline for up to 5 years
*Patients were administered VPRIV 60 U/kg every other week. N=39 at beginning of Study 044; n=34 at 12 months; n=38 at 24 months; n=37 at 36 months; n=30 at 48 months; and n=10 at 60 months.1,2

Mean Increase in Platelet Count from baseline1,2

+66% at 12 months in Study 032 (n=12) P=0.0023,4

  • Mean platelet count at baseline: 97 x 109/L
  • Mean change from baseline to 12 months for patients receiving the 60 U/kg dose:
    51 x 109/L ± 12 (standard error)

+120% at 24 months in Study 044 (n=37)1,2

  • Mean platelet count at baseline: 108.64 x 109/L
  • Mean change from baseline at 24 months: 87.85 x 109/L; maintained stability vs baseline for up to 5 years
Patients were administered VPRIV 60 U/kg every other week. N=39 at beginning of Study 044; n=32 at 12 months; n=37 at 24 months; n=34 at 36 months; n=29 at 48 months; and n=8 at 60 months.1,2

Mean Decrease in Spleen Volume from baseline1,2

-50% at 12 months in Study 032 (n=12) P=0.0033,4

  • Mean spleen volume at baseline: 2.9% of body weight
  • Mean change from baseline to 12 months for patients receiving the 60 U/kg dose: -1.9% ± 0.5 of body weight (standard error)

-64% at 24 months in Study 044 (n=30)1,2

  • Mean spleen volume at baseline: 3.847% of body weight
  • Mean change from baseline at 24 months: -2.662% of body weight; maintained stability vs baseline for up to 5 years
Patients were administered VPRIV 60 U/kg every other week. N=39 at beginning of Study 044; n=29 at 12 months; n=30 at 24 months; n=27 at 39 months; n=20 at 51 months; and n=9 at 63 months.1,2

Mean Decrease in Liver Volume from baseline1,2

-17% at 12 months in Study 032 (n=12) P=0.033,4

The decrease was not statistically significant after adjusting for multiple tests

  • Mean liver volume at baseline: 3.6% of body weight
  • Mean change from baseline to 12 months for patients receiving the 60 U/kg dose: -0.84% ± 0.33 of body weight (standard error)

-27% at 24 months in Study 044 (n=39)1,2§

  • Mean liver volume at baseline: 4.029% of body weight
  • Mean change from baseline at 24 months: -1.206% of body weight; maintained stability vs baseline for up to 5 years
§Patients were administered VPRIV 60 U/kg every other week. N=39 at beginning of Study 044; n=36 at 12 months; n=39 at 24 months; n=36 at 39 months; n=28 at 51 months; and n=9 at 63 months.1,2

Efficacy of VPRIV in Patients Previously Treated with Imiglucerase

In the overall population of treatment-naïve patients previously treated with imiglucerase from Study 039:

Mean Increase in Hemoglobin Concentration from baseline1,2

Through 9 months of treatment in Study 039 (n=17)3,5

  • Mean hemoglobin concentration at baseline: 11.0 g/dL
  • Mean treatment difference in change from baseline to 9 months: 0.1 g/dL ± 0.4 (standard error)

After 9 months of treatment, the mean absolute increase from baseline in hemoglobin concentration was 1.6 g/dL ± 0.2 (standard error)

+20% at 24 months in Study 044 (n=16)1,2||

  • Mean hemoglobin concentration at baseline: 10.58 g/dL
  • Mean change from baseline at 24 months: 2.00 g/dL; maintained stability vs baseline for up to 48 months
||Patients were administered VPRIV 60 U/kg every other week. N=16 at beginning of Study 044; n=15 at 9 months; n=14 at 12 months; n=16 at 24 months; n=10 at 36 months; and n=5 at 48 months.1,2

Mean Increase in Platelet count from baseline1,2

Through 9 months of treatment in Study 039 (n=17)3,5

  • Mean platelet count at baseline: 171 x 109/L
  • Mean treatment difference in change from baseline to 9 months: -39 x 109/L

+134% at 24 months in Study 044 (n=15)1,2¶

  • Mean platelet count at baseline: 186.25 x 109/L
  • Mean change from baseline at 24 months: 160.94 x 109/L; maintained stability vs baseline for up to 48 months
Patients were administered VPRIV 60 U/kg every other week. N=16 at beginning of Study 044; n=15 at 9 months; n=13 at 12 months; n=15 at 24 months; n=10 at 36 months; and n=5 at 48 months.1,2

Mean Decrease in Spleen Volume from baseline1,2

Through 9 months of treatment in Study 039 (n=7)3,5

  • Mean spleen volume at baseline: 3.4% of body weight
  • Mean treatment difference in change from baseline to 9 months: 0.1% of body weight

-64% at 24 months in Study 044 (n=6)1,2#

  • Mean spleen volume at baseline: 4.702% of body weight
  • Mean change from baseline at 24 months: -3.633% of body weight; maintained stability vs baseline for up to 51 months
#Patients were administered VPRIV 60 U/kg every other week. N=16 at beginning of Study 044; n=7 at 9 months; n=4 at 12 months; n=6 at 24 months; n=2 at 39 months; and n=2 at 51 months.1,2,5

Mean Decrease in Liver Volume from baseline1,2

Through 9 months of treatment in Study 039 (n=17)3,5

  • Mean liver volume at baseline: 4.3% of body weight
  • Mean treatment difference in change from baseline to 9 months: -0.1% of body weight

-37% at 24 months in Study 044 (n=15)1,2**

  • Mean liver volume at baseline: 4.212% of body weight
  • Mean change from baseline at 24 months: -1.688% of body weight; maintained stability vs baseline for up to 51 months
**Patients were administered VPRIV 60 U/kg every other week. N=16 at beginning of Study 044; n=17 at 9 months; n=12 at 12 months; n=15 at 24 months; n=9 at 39 months; and n=8 at 51 months.1,2,5

Efficacy of VPRIV in Patients Who Switched

In the overall population of patients who switched from imiglucerase to VPRIV from Study 034:

Change in Hemoglobin Concentration from baseline2,6

Through 12 months of treatment in Study 034 (n=40)3,7

  • Median hemoglobin concentration at baseline: 13.8 g/dL
  • Median hemoglobin concentration after 12 months: 13.5 g/dL

0% at 24 months in Study 044 (n=36)2,6††

  • Mean hemoglobin concentration at baseline: 13.82 g/dL
  • Mean change from baseline at 24 months: -0.05 g/dL; maintained stability vs baseline for up to 5 years
††Patients were administered VPRIV every other week at the same dose as their previous imiglucerase dose (15-60 U/kg). N=38 at beginning of Study 044; n=36 at 12 months; n=36 at 24 months; n=16 at 36 months; n=6 at 48 months; and n=6 at 60 months.2,6

Mean Increase in Platelet count from baseline2,6

Through 12 months of treatment in Study 034 (n=40)3,7

  • Median platelet count at baseline: 162 x 109/L
  • Median platelet count after 12 months: 174 x 109/L

+8% at 24 months in Study 044 (n=35)2,6‡‡

  • Mean platelet count at baseline: 164.5 x 109/L
  • Mean change from baseline at 24 months: 9.03 x 109/L; maintained stability vs baseline for up to 5 years
‡‡Patients were administered VPRIV every other week at the same dose as their previous imiglucerase dose (15-60 U/kg). N=38 at beginning of Study 044; n=36 at 12 months; n=35 at 24 months; n=16 at 36 months; n=6 at 48 months; and n=6 at 60 months.2,6

Mean Decrease in Spleen Volume from baseline2,6

Through 12 months of treatment in Study 034 (n=36)7

  • Mean percentage change from baseline to 12 months: -5.6%
  • Median spleen volume at baseline: 2.5 multiples of normal

— Within the predefined efficacy criterion of ±15%

-8% at 24 months in Study 044 (n=34)2,6§§

  • Mean spleen volume at baseline: 0.821% of body weight
  • Mean change from baseline at 24 months: -0.110% of body weight; maintained stability vs baseline for up to 5 years
§§Patients were administered VPRIV every other week at the same dose as their previous imiglucerase dose (15-60 U/kg). N=38 at beginning of Study 044; n=34 at 12 months; n=34 at 24 months; n=13 at 39 months; n=5 at 51 months; and n=4 at 63 months.2,6

Mean Decrease in Liver Volume from baseline2,6

Through 12 months of treatment in Study 034 (n=40)7

  • Median liver volume at baseline: 0.8 multiples of normal
  • Mean percentage change from baseline to 12 months: 0.0%

— Within the predefined efficacy criterion of ±15%

-1% at 24 months in Study 044 (n=37)2,6||||

  • Mean liver volume at baseline: 2.062% of body weight
  • Mean change from baseline at 24 months: -0.026% of body weight; maintained stability vs baseline for up to 5 years
||||Patients were administered VPRIV every other week at the same dose as their previous imiglucerase dose (15-60 U/kg). N=38 at beginning of Study 044; n=37 at 12 months; n=37 at 24 months; n=15 at 39 months; n=6 at 51 months; and n=5 at 63 months.2,6
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Important Safety Information

Hypersensitivity reactions, including anaphylaxis have occurred. The most serious adverse reactions in patients treated with VPRIV (velaglucerase alfa for injection) were hypersensitivity reactions.

Hypersensitivity reactions were the most commonly observed adverse reactions in patients treated with VPRIV in clinical studies. Patients were not routinely pre-medicated prior to infusion of VPRIV. The most commonly observed symptoms of hypersensitivity reactions were: headache, dizziness, hypotension, hypertension, nausea, fatigue/asthenia, and pyrexia. Hypersensitivity reactions in the clinical trials include any event considered related to and occurring within up to 24 hours of VPRIV infusion, including one case of anaphylaxis. Generally the reactions were mild and, in treatment-naïve patients, onset occurred mostly during the first 6 months of treatment and tended to occur less frequently with time.

As with any intravenous protein product, hypersensitivity reactions are possible, therefore appropriate medical support, including personnel adequately trained in cardiopulmonary resuscitative measures and access to emergency measures should be readily available when VPRIV is administered. If anaphylactic or other acute reactions occur, immediately discontinue the infusion of VPRIV and initiate the appropriate medical treatment.

Management of hypersensitivity reactions should be based on severity of the reaction, such as slowing the infusion rate, treatment with medications such as antihistamines, antipyretics and/or corticosteroids, and/or stopping and resuming treatment with increased infusion time. In cases where patients have exhibited symptoms of hypersensitivity to the active ingredient or excipients in the drug product or to other enzyme replacement therapy, pre-treatment with antihistamines and/or corticosteroids may prevent subsequent reactions.

The most common adverse reactions during clinical studies (in ≥10% of patients) were hypersensitivity reactions, headache, dizziness, abdominal pain, nausea, back pain, joint pain, prolonged activated PTT, fatigue/asthenia, and pyrexia. In clinical studies, the overall frequency of adverse events was generally higher in the population naïve to ERT (enzyme replacement therapy) than in the population switched from imiglucerase to VPRIV.

VPRIV is classified as pregnancy category B.

The safety and efficacy profiles were similar in pediatric (ages 4 to 17) and adult patients. The safety of VPRIV has not been established in patients under 4 years of age. Adverse reactions more commonly seen in pediatric patients compared to adult patients include (>10% difference): rash, aPTT prolonged, and pyrexia.

The adverse reaction profile in elderly patients was consistent with that previously observed across pediatric and adult patients. In general, dose selection for an elderly patient should be approached cautiously, considering comorbid conditions.

As with all therapeutic proteins, there is a potential for immunogenicity. In clinical studies, 1 of 54 enzyme treatment-naïve patients treated with VPRIV developed IgG class antibodies (neutralizing in an in vitro assay). It is unknown if the presence of IgG antibodies to VPRIV is associated with a higher risk of infusion reactions. Patients with an immune response to other enzyme replacement therapies who are switching to VPRIV should continue to be monitored for antibodies to VPRIV.

Please see Full Prescribing Information.

To report suspected adverse reactions, contact Shire Medical Information at 1-866-888-0660, option 2 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

For assistance with medical inquiries about VPRIV, please contact Medical Information at 1-866-888-0660, option 2 or email
US_ShireHGT_MedicalInformation@shire.com.

Indication

VPRIV® (velaglucerase alfa for injection) is indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease.

References

  1. Hughes DA, Gonzalez DE, Lukina EA, et al. Velaglucerase alfa (VPRIV) enzyme replacement therapy in patients with Gaucher disease: long-term data from phase III clinical trials. Am J Hematol. 2015;90(7):584-591, e1-e3.
  2. Data on file. Clinical Study Report: HGT-GCB-044. Shire, 2013; Lexington, MA.
  3. VPRIV [prescribing information]. Lexington, MA: Shire.
  4. Gonzalez DE, Ben Turkia H, Lukina EA, et al. Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: results from a randomized, double-blind, multinational, phase 3 study. Am J Hematol. 2013;88(3):166-171.
  5. Ben Turkia H, Gonzalez DE, Barton NW, et al. Velaglucerase alfa enzyme replacement therapy compared with imiglucerase in patients with Gaucher disease. Am J Hematol. 2013;88(3):179-184.
  6. Elstein D, Mehta A, Hughes DA, et al. Safety and efficacy results of switch from imiglucerase to velaglucerase alfa treatment in patients with type 1 Gaucher disease. Am J Hematol. 2015;90(7):592-597.
  7. Zimran A, Pastores GM, Tylki-Szymanska A, et al. Safety and efficacy of velaglucerase alfa in Gaucher disease type 1 patients previously treated with imiglucerase. Am J Hematol. 2013;88(3):172-178.