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IMPORTANT SAFETY INFORMATION FOR SOLIRIS® (eculizumab)

[injection, for intravenous use]

WARNING: SERIOUS MENINGOCOCCAL INFECTIONS

Life-threatening and fatal meningococcal infections have occurred in patients treated with Soliris®. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early [see Warnings and Precautions (5.1)].

  • Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccination in patients with complement deficiencies.
  • Immunize patients with meningococcal vaccines least 2 weeks prior to administering the first dose of Soliris®, unless the risks of delaying Soliris® therapy outweigh the risk of developing a meningococcal infection. [See Warnings and Precautions (5.1) for additional guidance on the management of the risk of meningococcal infection.]
  • Monitor patients for early signs of meningococcal infections and evaluate immediately if infection is suspected.

Soliris® is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Under the Soliris® REMS, prescribers must enroll in the program [see Warnings and Precautions (5.2)]. Enrollment in the Soliris REMS program and additional information are available by telephone: 1-888-SOLIRIS (1-888-765-4747) or at solirisrems.com.

Indications and Usage

Paroxysmal Nocturnal Hemoglobinuria (PNH)
Soliris® is indicated for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis.

Atypical Hemolytic Uremic Syndrome (aHUS)
Soliris® is indicated for the treatment of patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy.

Limitation of Use
Soliris® is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).

Contraindications

Soliris® is contraindicated in:

  • Patients with unresolved serious Neisseria meningitidis infection
  • Patients who are not currently vaccinated against Neisseria meningitidis, unless the risks of delaying Soliris® treatment outweigh the risks of developing a meningococcal infection

Warnings and Precautions

Other Infections
Soliris® blocks terminal complement activation; therefore patients may have increased susceptibility to infections, especially with encapsulated bacteria. Additionally, Aspergillus infections have occurred in immunocompromised and neutropenic patients. Children treated with Soliris® may be at increased risk of developing serious infections due to Streptococcus pneumoniae and Haemophilus influenza type b (Hib). Administer vaccinations for the prevention of Streptococcus pneumoniae and Haemophilus influenza type b (Hib) infections according to ACIP guidelines. Use caution when administering Soliris® to patients with any systemic infection.

Monitoring Disease Manifestations After Soliris® Discontinuation
Treatment Discontinuation for PNH
Monitor patients after discontinuing Soliris® for at least 8 weeks to detect hemolysis.

Treatment Discontinuation for aHUS
After discontinuing Soliris®, monitor patients with aHUS for signs and symptoms of thrombotic microangiopathy (TMA) complications for at least 12 weeks. In aHUS clinical trials, 18 patients (5 in the prospective studies) discontinued Soliris® treatment. TMA complications occurred following a missed dose in 5 patients, and Soliris® was reinitiated in 4 of these 5 patients.

Clinical signs and symptoms of TMA include changes in mental status, seizures, angina, dyspnea, or thrombosis. In addition, the following changes in laboratory parameters may identify a TMA complication: occurrence of two, or repeated measurement of any one of the following: a decrease in platelet count by 25% or more compared to baseline or the peak platelet count during Soliris® treatment; an increase in serum creatinine by 25% or more compared to baseline or nadir during Soliris® treatment; or, an increase in serum LDH by 25% or more over baseline or nadir during Soliris® treatment.

If TMA complications occur after Soliris® discontinuation, consider reinstitution of Soliris® treatment, plasma therapy [plasmapheresis, plasma exchange, or fresh frozen plasma infusion (PE/PI)], or appropriate organ-specific supportive measures.

Thrombosis Prevention and Management
The effect of withdrawal of anticoagulant therapy during Soliris® treatment has not been established. Therefore, treatment with Soliris® should not alter anticoagulant management.

Infusion Reactions
As with all protein products, administration of Soliris® may result in infusion reactions, including anaphylaxis or other hypersensitivity reactions. In clinical trials, no patients experienced an infusion reaction which required discontinuation of Soliris®. Interrupt Soliris® infusion and institute appropriate supportive measures if signs of cardiovascular instability or respiratory compromise occur.

Adverse Reactions

The most frequently reported adverse reactions in the PNH randomized trial (≥10% overall and greater than placebo) are: headache, nasopharyngitis, back pain, and nausea.

The most frequently reported adverse reactions in aHUS single-arm prospective trials (≥20%) are: headache, diarrhea, hypertension, upper respiratory tract infection, abdominal pain, vomiting, nasopharyngitis, anemia, cough, peripheral edema, nausea, urinary tract infections, pyrexia.

Please see full Prescribing Information for Soliris®, including boxed WARNING regarding serious meningococcal infection.

IMPORTANT SAFETY INFORMATION FOR STRENSIQ® (asfotase alfa)

[for injection]

Indications and Usage

STRENSIQ® is indicated for the treatment of patients with perinatal-, infantile- and juvenile-onset hypophosphatasia (HPP).

Warnings and Precautions

Hypersensitivity reactions, including anaphylaxis, have been reported in STRENSIQ®-treated patients. Signs and symptoms consistent with anaphylaxis included difficulty breathing, choking sensation, nausea, periorbital edema, and dizziness. These reactions have occurred within minutes after subcutaneous administration of STRENSIQ® and can occur in patients on treatment for more than one year. Other hypersensitivity reactions have also been reported in STRENSIQ®-treated patients, including vomiting, fever, headache, flushing, irritability, chills, skin erythema, rash, pruritus and oral hypoesthesia.  If a severe hypersensitivity reaction occurs, discontinue STRENSIQ® treatment and initiate appropriate medical treatment. Consider the risks and benefits of re-administering STRENSIQ® 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.

Localized lipodystrophy, including lipoatrophy and lipohypertrophy, has been reported at injection sites after several months in patients treated with STRENSIQ®. Advise patients to follow proper injection technique and to rotate injection sites.

Patients with HPP are at increased risk for developing ectopic calcifications. In clinical trials, 14 cases (14%) of ectopic calcification of the eye including the cornea and conjunctiva, and the kidneys (nephrocalcinosis) were reported. There was insufficient information to determine whether or not the reported events were consistent with the disease or due to STRENSIQ®. No visual changes or changes in renal function were reported.  Ophthalmology examinations and renal ultrasounds are recommended at baseline and periodically during treatment with STRENSIQ® to monitor for signs and symptoms of ophthalmic and renal ectopic calcifications and for changes in vision or renal function.

Adverse Reactions

The most common adverse reactions (≥ 10%) are injection site reactions, lipodystrophy, ectopic calcifications and hypersensitivity reactions.

Please see full Prescribing Information for STRENSIQ®.

IMPORTANT SAFETY INFORMATION FOR KANUMA® (sebelipase alfa)

[intravenous infusion]

Indications and Usage

KANUMA® is indicated for the treatment of patients with a diagnosis of lysosomal acid lipase deficiency (LAL-D).  

WARNINGS AND PRECAUTIONS

Hypersensitivity Reactions Including Anaphylaxis

Hypersensitivity reactions, including anaphylaxis, have been reported in KANUMA®-treated patients. In clinical trials, 3 of 106 (3%) patients treated with KANUMA® experienced signs and symptoms consistent with anaphylaxis. These patients experienced reactions during infusion with signs and symptoms including chest discomfort, conjunctival injection, dyspnea, generalized and itchy rash, hyperemia, swelling of eyelids, rhinorrhea, severe respiratory distress, tachycardia, tachypnea, and urticaria. Anaphylaxis has occurred as early as the sixth infusion and as late as 1 year after treatment initiation.

In clinical trials, 21 of 106 (20%) KANUMA®-treated patients, including 9 of 14 (64%) infants and 12 of 92 (13%) pediatric patients, 4 years and older, and adults experienced signs and symptoms either consistent with or that may be related to a hypersensitivity reaction. Signs and symptoms of hypersensitivity reactions, occurring in two or more patients, included abdominal pain, agitation, fever, chills, diarrhea, eczema, edema, hypertension, irritability, laryngeal edema, nausea, pallor, pruritus, rash, and vomiting. The majority of reactions occurred during or within 4 hours of the completion of the infusion. Patients were not routinely pre-medicated prior to infusion of KANUMA® in these clinical trials.

Due to the potential for anaphylaxis, appropriate medical support should be readily available when KANUMA® is administered.

Consider the risks and benefits of re-administering KANUMA® following a severe reaction. Monitor patients, with appropriate resuscitation measures available, if the decision is made to re-administer the product.

Hypersensitivity to Eggs or Egg Products

Patients with a known history of egg allergies were excluded from the clinical trials. Consider the risks and benefits of treatment with KANUMA® in patients with known systemic hypersensitivity reactions to eggs or egg products.

ADVERSE REACTIONS

The most common adverse reactions are:

In Patients with Rapidly Progressive Disease Presenting within the First 6 Months of Life (>30%): diarrhea, vomiting, fever, rhinitis, anemia, cough, nasopharyngitis, and urticaria.

In Pediatric and Adult Patients (≥8%): headache, fever, oropharyngeal pain, nasopharyngitis, asthenia, constipation, and nausea.

Please see full Prescribing Information for KANUMA®.