HAE treatment2019-11-25T08:24:59+02:00

WHAT IS HAE?

Approved HAE Treatments

Six products are currently approved and registered by the FDA (the United States Food and Drug Administration), the EMA (the European Medicines Agency) and a number of other countries to treat HAE:

Approved by: FDA EMA

Berinert®

brand of plasma derived C1-inhibitor. Berinert® is delivered intravenously and is approved for self-administration.
Company: CSL Behring

Yes Yes

Cinryze™

brand of plasma derived C1-inhibitor. Cinryze™ is delivered intravenously and is approved for self-administration.
Company: Takeda

Yes Yes

Firazyr®

brand of bradykinin receptor antagonist. Firazyr® is delivered by subcutaneous injection and is approved for self-administration. Company: Takeda

Yes Yes

HAEGARDA® — Berinert® 2000/3000

brand of plasma-derived C1 Esterase Inhibitor Subcutaneous (Human). HAEGARDA® is delivered by subcutaneous injection and is approved for self-administration. Company: CSL Behring

Yes Yes

Kalbitor®

brand of plasma kallikrein inhibitor. Kalbitor® is delivered through subcutaneous injections.
Company: Takeda

Yes Yes

Ruconest®

brand of recombinant C1-inhibitor. Ruconest® is delivered intrave-nously and is approved for self-administration.
Company: Pharming Group

Yes Yes

TAKHZYRO™ (lanadelumab)

brand of fully human monoclonal antibody providing targeted inhibition of plasma kallikrein. TAKHZYRO™ is delivered through subcutaneous self-injection.
Company: Takeda

Yes Yes

As might be expected from its different pathogenesis, angioedema seen in HAE does not respond to the drugs employed in treating other forms of urticaria/angioedema such as antihistamines, epinephrine, and corticosteroids. While epinephrine, in particular, may have a transient effect on swelling, it does not alter the course of an attack.

Maintaining airway patency is the primary concern for patients with laryngeal edema. If the airway is threatened, the patient should be intubated by an experienced physician. In addition, the capability for emergency tracheostomy should be readily available. Because gastrointestinal edema usually involves excruciating pain, frequent vomiting, and the potential for hypotension, therapy should include aggressive fluid replacement and pain management.
In the past, some physicians have used fresh frozen plasma in the acute attack setting, but this therapy is considered controversial because in addition to C1-inhibitor, fresh frozen plasma contains substrates of the complement and kinin systems that could produce a vasoactive peptide and cause an attack exacerbation.

Treating Children with HAE

Fortunately, most prepubescent children with HAE do not suffer from frequent attacks and infrequent flares affecting the abdomen can be managed by using pain relievers and anti nausea agents. The small number of severely affected children who experience frequent and severe attacks must be managed on a case by case basis. Every physician will need to discern for him or herself how the approved medications may prove most useful for treating their young patients.

New Treatments on the Horizon

New HAE medicines are still being developed:

  • Adverum – ADVM-053: A gene therapy candidate being investigated as a potential single‑administration treatment to provide sustained levels of the C1 esterase inhibitor protein – learn more
  • Alnylam Pharmaceuticals – AKN-F12: A RNA interference (RNAi) drug to knockdown factor XII – learn more
  • Arrowhead – ARC-F12: An RNAi therapeutic inhibiting the production of Factor XII – learn more
  • Attune Pharmaceuticals – ATN-249: An orally-administered plasma kallikrein inhibitor – learn more
  • BioCryst – BCX7353: A once-daily oral treatment (capsule or liquid) inhibiting plasma kallikrein – learn more
  • CSL Behring – CSL312: A humanised anti-factor XIIa monoclonal antibody for subcutaneous treatment – learn more
  • Ionis – IONIS-PKK-LRx: A Generation 2+ ligand-conjugated antisense drug designed to reduce prekallikrein production – learn more
  • KalVista – KVD824: An oral plasma kallikrein inhibitor – learn more
  • KalVista – KVD900: An oral plasma kallikrein inhibitor – learn more
  • Pharvaris – PHA121: An orally bioavailable small-molecule antagonizing the B2 receptor – learn more
  • REGENXBIO: A gene therapy product candidate utilizing NAV Vectors designed to deliver a gene encoding a therapeutic antibody targeting and binding to plasma kallikrein – learn more
  • Verseon – an oral, small-molecule plasma kallikrein inhibitors – learn more
STAY TUNED

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Anthony J. Castaldo, President, HAEi

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