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Excellence in Rare Diseases or Orphan Drugs

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Finalist

Introducing the First Treatment Option for Paediatric and Adult Patients with Primary HLH

by Sobi, Inc. (North America)
with support from W2O Group


Summary of work

Primary haemophagocytic lymphohistiocytosis (HLH), which typically develops within the first year of life, is an ultra-rare syndrome of hyperinflammation where an overexpression of interferon gamma (IFNγ) can cause the immune system to attack the body. If left untreated, primary HLH can be fatal.

Gamifant® (emapalumab-lzsg) – the first and only medicine approved in the US to treat patients with primary HLH – is a critical advancement for patients with high mortality. Approved by the US FDA in late 2018, Gamifant’s targeted treatment approach can be used to bring hyperinflammatory emergencies under control and prepare patients for a haematopoietic stem cell transplant (HSCT).

Today, Sobi remains committed to raising awareness of primary HLH and its symptoms, and continues to lead and execute healthcare provider, advocacy and caregiver initiatives and programmes.

Judges' comments

This entry was particularly well-structured and there was a real sense of difference within the initiative. They really identified the unmet needs of patients and families with Primary HLH.


Winner

Transforming Care in Hereditary Angioedema - by Takeda UK
with support from Makara Health


Finalists

Transforming the Rare Disease Sales Call - by Sanofi Genzyme
with support from Redline Strategic

Introducing the First Treatment Option for Paediatric and Adult Patients with Primary HLH - by Sobi, Inc. (North America)
with support from W2O Group


PMEA Awards 2020

Key dates

Entry deadline 27 August 2020
Extended entry deadline 3 September 2020*
*additional fee applies
Judging Day 1 October 2020
Company of the Year Interviews 22 October 2020
PMEA Event 25 November 2020