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Pharming Juli-2021

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Takeda Q1 (April 2021-March 2022) HAE highlights

Revenue in Hereditary Angioedema (“HAE”) was 39.0 billion JPY, a year-on-year increase of 0.7 billion JPY, or 1.8%.
Sales of TAKHZYRO were 25.5 billion JPY, an increase of 2.2 billion JPY, or 9.6%, versus the same period of the
previous fiscal year primarily due to new launches including prefilled syringe administration in Europe. Sales of FIRAZYR
decreased by 1.2 billion JPY, or 15.1%, to 6.9 billion JPY, primarily due to the continued impact of generic entrants in the

Rare Genetics & Hematology
In Rare Genetics & Hematology, Takeda focuses on hereditary angioedema to transform the treatment paradigm, including
through TAKHZYRO, and on rare hematology and rare metabolic diseases, with the aim to deliver functional cures in a
select group of diseases using novel modalities and platforms.

TAKHZYRO / Generic name: lanadelumab
– In July 2021, Takeda announced the results from two final analyses from the Phase 3 HELP (Hereditary
Angioedema Long-term Prophylaxis) Study™ Open-label Extension (OLE), which evaluated the long-term safety
(primary endpoint) and efficacy of TAKHZYRO (lanadelumab) 300 mg every two weeks for up to 2.5 years. In the
first analysis, the mean (min, max) reduction in the attack rate compared to baseline observed in the study
population (N=212) was of 87.4 percent (-100; 852.8), and the median reduction was 97.7 percent and patients
received treatment for a mean (standard deviation) duration of 29.6 (8.2) months. At steady state – day 70 to the end
of the treatment period – attack rates were further reduced to a mean of 92.4 percent and a median reduction of 98.2
percent. An additional analysis further suggests TAKHZYRO was a well-tolerated treatment that prevented HAE
attacks over an extended planned 132 week treatment period across specific HAE patient demographic and disease
characteristic subgroups. These data were presented at the 2021 European Academy of Allergy and Clinical
Immunology (EAACI) Hybrid Congress.
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