January 22, 2021

Japan’s regulator approves Grifols’ Lynspad™ [alpha-1 proteinase inhibitor] for the treatment of alpha-1 antitrypsin deficiency

  • Grifols collaborating with Japan’s OrphanPacific, Inc., which facilitated regulatory approval and will commercialize Lynspad™
  • Lynspad™ (intravenous infusion 1000 mg) is used to treat patients diagnosed with severe alpha-1 antitrypsin deficiency

Barcelona, Spain, and Tokyo, January 22, 2021 - Grifols (MCE:GRF, MCE:GRF.P, NASDAQ:GRFS), one of the world’s leading producers of plasma-derived medicines with a more than 100-year track record of contributing to the health and well-being of people around the world, and OrphanPacific, Inc., which provides drugs for rare diseases and disorders in Japan, today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted manufacturing and marketing approval for Lynspad™ (intravenous infusion 1000 mg), an alpha-1 proteinase inhibitor to treat severe alpha-1 antitrypsin deficiency (AATD) in Japan.

Grifols developed Lynspad™ (sold as Prolastin-C® in other markets) and designated OrphanPacific as the Appointed Marketing Authorization Holder (AMAH) to obtain manufacturing and marketing approval for the AATD treatment in Japan under the Foreign Exceptional Approval System, an approach for companies looking to commercialize an overseas-manufactured product in Japan without a license to manufacture and distribute pharmaceutical products in the country.

OrphanPacific will also support Grifols in discussion with the MHLW to establish Lynspad’s™ pricing within the country’s National Health Insurance system, as well as with the commercialization of the treatment.

The collaboration between Grifols and OrphanPacific is the result of the Innovative Pharma Model (IPM) that CMIC Group created to support the market-entry strategy of foreign pharmaceutical companies in Japan. OrphanPacific is a joint-venture of CMIC Group, a leading contract research organization, and MEDIPAL, a leading pharmaceutical wholesale distributor in the country.

As a pharmaceutical business license organization in Japan, OrphanPacific delivers the IPM and provides full pharmaceutical-firm capabilities that enable international companies to introduce their innovative treatments in the Japanese market.

Lynspad™ is used to treat patients diagnosed with severe AATD [serum alpha-1 antitrypsin level < 50 mg/dL (measured by nephelometry)] and with pulmonary disease such as chronic obstructive pulmonary disease (COPD) and emphysema accompanied by airflow obstruction.

In patients with AATD, serum and tissue levels of alpha-1 proteinase inhibitor are reduced, resulting in an imbalance between neutrophil elastase and its inhibitor, alpha-1 proteinase inhibitor. The imbalance causes inappropriate proteolysis in lung tissue, but Lynspad™ augmentation therapy enhances protection against proteinases by increasing and maintaining the level of alpha-1 proteinase inhibitor in serum and pulmonary airway epithelial lining fluid, and corrects proteinase vs. inhibitor imbalance. It is believed that correction of this imbalance suppresses the onset and progression of emphysema and delays the progression of pathological condition of COPD.

Summary of Approval Details:

Product Name Lynspad™ for Intravenous Infusion 1000 mg
Non-proprietary name Alpha-1 Proteinase Inhibitor (Human)
Indication Severe alpha-1 antitrypsin deficiency
Dosage and administration For adults, 60 mg/kg is normally intravenously infused once a week as Alpha1-Proteinase Inhibitor (Human)
Foreign Exceptional Approval Holder Grifols Therapeutics LLC

Marketing Authorization Holder (AMAH) in Japan
(Appointed Manufacturer /Distributor of Foreign-manufactured Pharmaceutical Product)

OrphanPacific, Inc.