Meet Jenn

A woman living with cystinosis, a rare metabolic disease that can damage many organs and tissues, particularly the kidneys and eyes.

INDICATION

CYSTARAN (CYSTEAMINE OPHTHALMIC SOLUTION) 0.44% STERILE is a cystine-depleting agent indicated for the treatment of corneal cystine crystal accumulation in patients with cystinosis.

IMPORTANT SAFETY INFORMATION

To minimize contaminating the dropper tip and solution, care should be taken not to touch the eyelids or surrounding areas with the dropper tip of the bottle. Keep bottle tightly closed when not in use.

There have been reports of benign intracranial hypertension (or pseudotumor cerebri) associated with oral cysteamine treatment that has resolved with the addition of diuretic therapy. There have also been reports associated with ophthalmic use of cysteamine; however, all of these patients were on concurrent oral cysteamine.

CYSTARAN contains benzalkonium chloride, which may be absorbed by soft contact lenses. Contact lenses should be removed prior to application of solution and may be reinserted 15 minutes following its administration.

CYSTARAN is for topical ophthalmic use only.

The most frequently reported ocular adverse reactions occurring in ≥ 10% of patients were sensitivity to light, redness, and eye pain/irritation, headache and visual field defects.

Please see full prescribing information for contraindications, precautions and adverse reactions.

Meet Ashley

A young woman living with ADA-SCID, a severe form of a rare, inherited disorder that damages the immune system.

INDICATION

Adagen® (pegademase bovine) Injection is indicated for enzyme replacement therapy for adenosine deaminase (ADA) deficiency in patients with severe combined immunodeficiency disease (SCID) who are not suitable candidates for – or who have failed – bone marrow transplantation.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

There is no evidence to support the safety and efficacy of Adagen as preparatory or support therapy for bone marrow transplantation. Since Adagen is administered by intramuscular injection, it should be used with caution in patients with thrombocytopenia and should not be used if thrombocytopenia is severe.

PRECAUTIONS

General

  • Any laboratory or clinical indication of a decrease in potency of Adagen should be reported immediately by telephone to Leadiant Biosciences, Inc. Tel. 1-866-792-5172.
  • Appropriate care to protect immune deficient patients should be maintained until improvement in immune function has been documented. The degree of immune function improvement may vary from patient to patient and, therefore, each patient will require appropriate care consistent with immunologic status.

Laboratory Tests

  • The treatment of SCID associated with ADA deficiency with Adagen should be monitored by measuring plasma ADA activity and red blood cell dATP levels.
  • Once treatment with Adagen has been initiated, a desirable range of plasma ADA activity (trough level before maintenance injection) should be 15-35 µmol/hr/mL and after 2 months of maintenance treatment with Adagen, red cell dATP levels should decrease to a range of ≤ 0.005 to 0.015 µmol/mL.
  • Failure to maintain adequate levels of plasma ADA activity in patients undergoing treatment with Adagen may lead to decline in immune function and increase risk of opportunistic infections.

Drug Interactions

  • There are no known drug interactions with Adagen. However, Vidarabine is a substrate for ADA and 2-deoxycoformycin is a potent inhibitor of ADA.

ADVERSE REACTIONS

  • Clinical experience with Adagen has been limited. The following adverse reactions were reported during clinical trials: headache in one patient and pain at the injection site in two patients.
  • The following adverse reactions have been identified during post-approval use of Adagen: hemolytic anemia, auto-immune hemolytic anemia, thrombocythemia, thrombocytopenia, autoimmune thrombocytopenia, lymphomas, injection site erythema, and urticaria.

To report SUSPECTED ADVERSE REACTIONS, contact Leadiant Biosciences, Inc. at 1-888-393-4584 or by email at drugsafety@leadiant.com or contact the FDA at 1-800-FDA-1088 or www.fda.gov/safety/medwatch.

Please see full prescribing information for contraindications, precautions and adverse reactions.

Meet the Monaco Family

One family, two children born with secondary carnitine deficiency, two drastically different outcomes. Learn about the critical role of newborn screening and how this family is making a difference in the lives of other families affected by carnitine deficiency and other rare diseases.

INDICATIONS

CARNITOR® (levocarnitine) Tablets, Oral Solution, and Sugar-Free Oral Solution are indicated for the treatment of primary systemic carnitine deficiency.

CARNITOR® (levocarnitine) Injection is indicated for the prevention and treatment of carnitine deficiency in patients with end stage renal disease who are undergoing dialysis.

CARNITOR® (levocarnitine) Injection, Tablets, Oral Solution, and Sugar-Free Oral Solution are also indicated for the acute and chronic treatment of patients with an inborn error of metabolism which results in secondary carnitine deficiency.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

None known.

WARNINGS

CARNITOR® (levocarnitine) Tablets, Oral Solution, and Sugar-Free Oral Solution

Hypersensitivity Reactions
Serious hypersensitivity reactions, including rash, urticaria, and facial edema have been reported with oral CARNITOR®. Other serious hypersensitivity reactions, including anaphylaxis, laryngeal edema, and bronchospasm have been reported following intravenous levocarnitine administration, mostly in patients with end stage renal disease undergoing dialysis.

Discontinue use of CARNITOR® and instruct patients to seek medical attention if they experience symptoms suggestive of a hypersensitivity reaction.

CARNITOR® (levocarnitine) Injection

Hypersensitivity Reactions
Serious hypersensitivity reactions, including anaphylaxis, laryngeal edema, and bronchospasm have been reported following CARNITOR® administration, mostly in patients with end stage renal disease who are undergoing dialysis. Some reactions occurred within minutes after intravenous administration of CARNITOR®.

If a severe hypersensitivity reaction occurs, discontinue CARNITOR® treatment and initiate appropriate medical treatment. Consider the risks and benefits of re-administering CARNITOR® to individual patients following a severe reaction. If the decision is made to re-administer the product, monitor patients for a reoccurrence of signs and symptoms of a severe hypersensitivity reaction.

PRECAUTIONS

General

The safety and efficacy of oral levocarnitine has not been evaluated in patients with renal insufficiency. Chronic administration of high doses of oral levocarnitine in patients with severely compromised renal function or in ESRD patients on dialysis may result in accumulation of the potentially toxic metabolites, trimethylamine (TMA) and trimethylamine-N-oxide (TMAO), since these metabolites are normally excreted in the urine.

CARNITOR® (levocarnitine) Oral Solution and CARNITOR® SF (levocarnitine) Sugar-Free Oral Solution are for oral/internal use only.

Drug Interactions

Reports of INR increase with the use of warfarin have been observed. It is recommended that INR levels be monitored in patients on warfarin therapy after the initiation of treatment with levocarnitine or after dose adjustments.

Carnitor® (levocarnitine) Tablets, Oral Solution, and Sugar-Free Oral Solution – Not for parenteral use

Gastrointestinal reactions may result from a too rapid consumption of carnitine. CARNITOR® (levocarnitine) Oral Solution and CARNITOR®SF (levocarnitine) Sugar-Free Oral Solution may be consumed alone, or dissolved in drinks or other liquid foods to reduce taste fatigue. They should be consumed slowly and doses should be spaced evenly throughout the day to maximize tolerance.

ADVERSE REACTIONS

Neurologic Reactions:Seizures have been reported to occur in patients, with or without pre-existing seizure activity, receiving either oral or intravenous levocarnitine. In patients with pre-existing seizure activity, an increase in seizure frequency and/or severity has been reported

Carnitor® (levocarnitine) Tablets, Oral Solution, and Sugar-Free Oral Solution

The following adverse reactions associated with the use of oral formulations of levocarnitine were identified in clinical trials or postmarketing reports. Because these reactions were reported voluntarily from a population of uncertain size, it is not always possible to estimate their frequency, reliability, or to establish a causal relationship to drug exposure.

Gastrointestinal Reactions:Various mild gastrointestinal complaints have been reported during the long-term administration of oral L- or D,L-carnitine; these include transient nausea and vomiting, abdominal cramps, and diarrhea. Gastrointestinal adverse reactions with CARNITOR® (levocarnitine) Oral Solution or CARNITOR® SF (levocarnitine) Sugar-Free Oral Solution dissolved in liquids might be avoided by a slow consumption of the solution or by a greater dilution. Decreasing the dosage often diminishes or eliminates drug-related patient body odor or gastrointestinal symptoms when present. Tolerance should be monitored very closely during the first week of administration, and after any dosage increases.

Musculoskeletal Reactions:Mild myasthenia has been described only in uremic patients receiving D,L-carnitine.

Hypersensitivity Reactions:Rash, urticaria, and facial edema have been reported with oral CARNITOR® (see WARNINGS).

Carnitor® (levocarnitine) Injection

Gastrointestinal Reactions:Transient nausea and vomiting have been observed. Less frequent adverse reactions are body odor, nausea, and gastritis. An incidence for these reactions is difficult to estimate due to the confounding effects of the underlying pathology.

Hypersensitivity Reactions:Anaphylaxis, laryngeal edema and bronchospasm (see WARNINGS).

To report SUSPECTED ADVERSE REACTIONS, contact Leadiant Biosciences, Inc. at 1-888-393-4584 or by email at drugsafety@leadiant.com or contact the FDA at 1-800-FDA-1088 or www.fda.gov/safety/medwatch.

Please see full prescribing information for contraindications, precautions and adverse reactions.
Click here for Carnitor® (levocarnitine) Injection full prescribing information

Click here for important Healthcare Professional information about Carnitor® (levocarnitine) Injection

Click here for Carnitor® (levocarnitine) Tablets, Oral Solution, and Sugar-Free Oral Solution full prescribing information

Click here for important Healthcare Professional information about Carnitor® Tablets, Oral Solution and Sugar-Free Oral Solution