Date: 2017-06-22
Type of information: Granting of a Market Authorisation in the US
Product name: Haegarda®
Compound: human C1 esterase Inhibitor
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases
Action mechanism:
- protein Haegarda® is a human plasma-derived, purified, pasteurized, lyophilized (freeze-dried) concentrate prepared from large pools of human plasma from U.S. donors. This self-administered, plasma-derived concentrate of C1 esterase inhibitor (C1-INH) is injected twice weekly subcutaneously. It targets the root cause of HAE by replacing deficient or dysfunctional natural C1-INH, restoring functional C1-INH levels to above 40 percent of normal levels, a percentage thought to be associated with reduced risk for HAE attacks.
Company: CSL (Australia)
Disease: prevention of hereditary angioedema (HAE) attacks in adolescent and adult patients
Latest news:
- • On June 22, 2017, the FDA approved Haegarda®, the first C1 esterase inhibitor for subcutaneous administration to prevent hereditary angioedema (HAE) attacks in adolescent and adult patients. The subcutaneous route of administration allows for easier at-home self-injection by the patient or caregiver, once proper training is received.
- The efficacy of Haegarda® was demonstrated in a multicenter controlled clinical trial. The study included 90 subjects ranging in age from 12 to 72 years old with symptomatic HAE. Subjects were randomized to receive twice per week subcutaneous doses of either 40 IU/kg or 60 IU/kg, and the treatment effect was compared to a placebo treatment period. During the 16 week treatment period, patients in both treatment groups experienced a significantly reduced number of HAE attacks compared to their placebo treatment period.
- The most common side effects included injection site reactions, hypersensitivity (allergic) reactions, nasopharyngitis (swelling of the nasal passages and throat) and dizziness. Haegarda should not be used in individuals who have experienced life-threatening hypersensitivity reactions, including anaphylaxis, to a C1-INH preparation or its inactive ingredients.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization: 2017-06-22
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA:
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
- • On,October 11, 2018, the Institute for Clinical and Economic Review (ICER) released an Evidence Report assessing the comparative clinical effectiveness and value of therapies for long-term prophylaxis against hereditary angioedema (HAE) attacks. The report reviews three therapies for the prevention of HAE attacks: lanadelumab (Takhzyro™, Shire Plc), and two C1 inhibitors (Haegarda®, CSL Behring ; and Cinryze®, Shire). ICER's earlier draft report also included an additional C1 inhibitor, Ruconest® (Pharming Healthcare,), which has since been removed from the assessment because the treatment is no longer under consideration for FDA approval for long-term prophylaxis.
- All three drugs reviewed reduced the number and severity of HAE attacks compared with no long-term prophylaxis, and available data suggest few harms. Haegarda and lanadelumab have the additional benefit of being subcutaneously administered, which may decrease the burden and complexity of administration and avoid complications due to repeated intravenous infusions. Evidence provides high certainty that Haegarda and Cinryze provide a substantial net benefit compared with no prophylaxis. Evidence on lanadelumab was considered promising but inconclusive because of concerns about long-term safety with a new therapy that was only studied in short-term trials and small numbers of patients. Evidence was insufficient to compare the net health benefits among the three treatments.
- Economic analyses assessing long-term cost-effectiveness found that all three treatments far exceed commonly cited thresholds of $50,000-$150,000 per quality-adjusted life year (QALY) gained, with $243,000 per QALY for Haegarda, $5,870,000 per QALY for Cinryze, and $1,020,000 per QALY for lanadelumab. To align costs with the added benefits for patients, discounts off the list price would need to be approximately 59% for Cinryze, 27% for Haegarda, and 33% for lanadelumab.
- However, because the overall cost-effectiveness of prophylactic treatment balances the high treatment cost of prophylaxis with the avoided high costs associated with on-demand treatment of acute attacks, the economic modeling results are highly sensitive to assumptions made about variables such as the baseline rate of acute attacks and the likelihood that patients will switch dosing schedules over time for prophylactic therapy.
Is general: Yes
