Rare Disease Therapeutics, Inc.

Rare Disease Therapeutics, Inc. Drugs

• Anascorp
  

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  Anascorp

  

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  For Intravenous use only.

  Initiate treatment with Anascorp as soon as possible after scorpion sting in patients who develop clinically important signs of scorpion envenomation, including but not limited to loss of muscle control, roving or abnormal eye movements, slurred speech, respiratory distress, excessive salivation, frothing at the mouth and vomiting.(2)

  Initial Dose: 3 vials

  The initial dose of Anascorp is 3 vials. Reconstitute the contents of each vial with 5 milliliters of sterile normal saline and mix by continuous gentle swirling. Combine the contents of the reconstituted vials promptly and further dilute to a total volume of 50 milliliters with sterile normal saline. Inspect the solution visually for particulate matter and discoloration prior to administration. Do not use if turbid. Infuse intravenously over 10 minutes. Monitor patient closely during and up to 60 minutes following the completion of infusion to determine if clinically important signs of envenomation have resolved. Discard partially used vials.

  Initial Dose: 3 vials

  Additional doses may be used if needed. Infuse one vial at a time at intervals of 30 to 60 minutes. Reconstitute the contents with 5 milliliters of sterile normal saline and mix by continuous gentle swirling. Further dilute to a total volume of 50 milliliters with sterile normal saline. Inspect the solution visually for particulate matter or discoloration prior to administration. Infuse intravenously over 10 minutes. Monitor patient closely during and up to 60 minutes following the completion of infusion to determine if clinically important signs of envenomation have resolved. Discard partially used vials.

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• Orfadin
  

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  Orfadin

  

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  2.1 Dosage

  The recommended dose of Orfadin is 1 to 2 mg/kg divided into two daily doses. The initial dose is 1 mg/kg/day divided for morning and evening administration. Treatment with Orfadin should be initiated by a physician experienced in the treatment of HT-1.

  The dose of Orfadin may be adjusted in each patient. In patients whose erythrocyte PBG-synthase activity and urine 5-ALA and urine succinylacetone are not normalized within one month after the start of Orfadin treatment, the Orfadin dose may be increased to 1.5 mg/kg/day. In patients receiving 1.5 mg/kg/day, whose erythrocyte PBG-synthase activity and urine 5-ALA and urine succinylacetone remain elevated and whose plasma succinylacetone is not normalized after three months, the dose may be increased to up a maximum dose of 2 mg/kg/day.

  If plasma nitisinone concentration, plasma succinylacetone, urine 5-ALA and erythrocyte PBG-synthase activity are not available, clinical laboratory assessments should include urine succinyl acetone, liver function tests, alpha fetoprotein, and serum tyrosine and phenylalanine level. During initiation of therapy and during acute exacerbations, it may be necessary to follow more closely all available biochemical parameters [see Warnings and Precautions (5)].

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• Cystadane
  

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  Cystadane

  

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  2.1 Dosage

  The usual dosage in adult and pediatric patients is 6 grams per day administered orally in divided doses of 3 grams twice daily.  In pediatric patients less than 3 years of age, dosage may be started at 100 mg/kg/day divided in twice daily doses, and then increased weekly by 50 mg/kg increments.  Therapy with Cystadane should be directed by physicians knowledgeable in the management of patients with homocystinuria.  Patient response to Cystadane can be monitored by homocysteine plasma levels.  Dosage in all patients can be gradually increased until plasma total homocysteine is undetectable or present only in small amounts. Response (by homocysteine plasma levels) usually occurs within several days and steady state within a month.  Plasma methionine concentrations should be monitored in patients with CBS deficiency [See Warnings and Precautions (5.1)].

  Dosages of up to 20 grams per day have been necessary to control homocysteine levels in some patients.  However, one pharmacokinetic and pharmacodynamic in vitro simulation study indicated minimal benefit from exceeding a twice-daily dosing schedule and a 150 mg/kg/day dosage for Cystadane.

  2.2 Administration

  The prescribed amount of Cystadane should be measured with the measuring scoop provided (one level 1.7 mL scoop is equal to 1 gram of betaine anhydrous powder) and then dissolved in 4 to 6 ounces (120 to 180 mL) of water, juice, milk, or formula, or mixed with food for immediate ingestion.

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