Cystadane (Page 2 of 2)

12.3 Pharmacokinetics

Pharmacokinetic studies of Cystadane are not available. Plasma levels of Cystadane have not been measured in patients and have not been correlated to homocysteine levels.

13 NONCLINICAL TOXICOLOGY

13.1 Carcinogenesis, Mutagenesis, Impairment of Fertility

Long-term carcinogenicity and fertility studies have not been conducted with Cystadane. No evidence of genotoxicity was demonstrated in the following tests: metaphase analysis of human lymphocytes; bacterial reverse mutation assay; and mouse micronucleus test.

14 CLINICAL STUDIES

Cystadane was studied in a double-blind, placebo-controlled, crossover study in 6 patients with CBS deficiency, ages 7 to 32 years at enrollment. Cystadane was administered at a dosage of 3 grams twice daily, for 12 months. Plasma homocystine levels were significantly reduced (p<0.01) compared to placebo. Plasma methionine levels were variable and not significantly different compared to placebo. No adverse events were reported in any patient.

Cystadane has also been evaluated in observational studies without concurrent controls in patients with homocystinuria due to CBS deficiency, MTHFR deficiency, or cbl defect.A review of 16 case studies and the randomized controlled trial previously described was also conducted, and the data available for each study were summarized; however, no formal statistical analyses were performed. The studies included a total of 78 male and female patients with homocystinuria who were treated with Cystadane. This included 48 patients with CBS deficiency, 13 with MTHFR deficiency, and 11 with cbl defect, ranging in age from 24 days to 53 years. The majority of patients (n=48) received 6 gm/day, 3 patients received less than 6 gm/day, 12 patients received doses from 6 to 15 gm/day, and 5 patients received doses over 15 gm/day. Most patients were treated for more than 3 months (n=57) and 30 patients were treated for 1 year or longer (range 1 month to 11 years). Homocystine is formed nonenzymatically from two molecules of homocysteine, and both have be used to evaluate the effect of Cystadane in patients with homocystinuria. Plasma homocystine or homocysteine levels were reported numerically for 62 patients, and 61 of these patients showed decreases with Cystadane treatment. Homocystine decreased by 83-88% regardless of pre-treatment level, and homocysteine decreased by 71-83%, regardless of the pre-treatment level. Clinical improvement, such as improvement in seizures, or behavioral and cognitive functioning, was reported by the treating physicians in about three-fourths of patients. Many of these patients were also taking other therapies such as vitamin B6 (pyridoxine), vitamin B12 (cobalamin), and folate with variable biochemical responses. In most cases, adding Cystadane resulted in a further reduction of either homocystine or homocysteine.

16 HOW SUPPLIED/STORAGE AND HANDLING

Cystadane is available in plastic bottles containing 180 grams of betaine anhydrous. Each bottle is equipped with a plastic child-resistant cap and is supplied with a polystyrene measuring scoop. One level scoop (1.7 mL) is equal to 1 gram of betaine anhydrous powder.

NDC 66621-4000-1 180 g/bottle

Cystadane can be ordered by calling AnovoRx Group, LLC, Customer service at 1-888-487-4703

16.1 Storage

Store at room temperature, 15 – 30 ˚C (59 – 86 ˚F). Protect from moisture.

17 PATIENT COUNSELING INFORMATION

Patients should be advised of the following information before beginning treatment with Cystadane:

17.1 Dosing and Administration

  • Instruct patients and caregivers that Cystadane should only be taken as directed by their healthcare professional.
  • Instruct patients and caregivers to administer Cystadane as follows:
  • Shake bottle lightly before removing cap.

— Measure with the scoop provided.

— Measure the number of scoops as prescribed by their healthcare professional. One level scoop (1.7 mL) is equivalent to 1 gram of betaine anhydrous powder.

— Mix powder with 4 to 6 ounces (120 to 180 mL) of water, juice, milk, or formula until completely dissolved, or mix with food, then ingest mixture immediately.

— Always replace the cap tightly after using, and protect powder from moisture.

Manufactured For:
Rare Disease Therapeutics, Inc.
Franklin, TN 37067

Under License From:
Orphan Europe, s.a.r.l. Puteaux France

Distributed By:
AnovoRx Distribution, LLC
Memphis, TN 38134
Part No.: RDT C PI007
Part No.: Orphan Europe OEP 829

Cystadane (betaine anhydrous) for oral solution
(click image for full-size original)
CYSTADANE betaine powder, for solution
Product Information
Product Type HUMAN PRESCRIPTION DRUG Item Code (Source) NDC:66621-4000
Route of Administration ORAL DEA Schedule
Active Ingredient/Active Moiety
Ingredient Name Basis of Strength Strength
BETAINE (BETAINE) BETAINE 6 g
Product Characteristics
Color white (none) Score
Shape Size
Flavor Imprint Code
Contains
Packaging
# Item Code Package Description Multilevel Packaging
1 NDC:66621-4000-1 180 POWDER, FOR SOLUTION (SCOOPFUL) in 1 BOTTLE None
Marketing Information
Marketing Category Application Number or Monograph Citation Marketing Start Date Marketing End Date
NDA NDA020576 10/25/1996
Labeler — Rare Disease Therapeutics, Inc. (966133100)
Registrant — Rare Disease Therapeutics, Inc. (966133100)
Establishment
Name Address ID/FEI Operations
Rare Disease Therapeutics, Inc. 966133100 relabel (66621-4000)
Establishment
Name Address ID/FEI Operations
Ropack Pharmaceutical 209989631 manufacture (66621-4000)

Revised: 01/2015 Rare Disease Therapeutics, Inc.

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