Targeting Rare Diseases 
and Critical Care

Dr. Rudolf Widmann established a successful innovative pharmaceutical company focussed on the treatment of rare diseases in 1996 in Vienna, Austria.

European Parliament adopted Regulation EC No 141/2000 The Orphan Regulation of Dec 16th, 1999. The Regulation lays down the EU procedure for the designation of orphan medicines, defines incentives for the development and placing of designated orphan medicines in the market, and establishes the Committee for Orphan Medicinal Products (COMP).

First marketing authorization for Anagrelide in Austria for treatment of patients with essential thrombocythemia.

The European Union (EU) has expanded several times throughout its history via the accession of new member states. The EU expansion towards Eastern and Central Europe and the Western Balkans had a positive influence on the territorial expansion of AOP Health as well, creating a basis for providing treatments for rare diseases in all AOP Health core markets.

Subcutaneous prostacyclin analogue approved for the treatment of pulmonary arterial hypertension (PAH) in Europe.

The Janus Kinase 2 gene (JAK2) was discovered in 2005. It provides instructions to cells for making the JAK2 protein. The test looks for mutations in JAK2 that are associated with bone marrow disorders caused by the production of too many blood cells.

IFNα has been used to treat malignant and viral disorders for more than 25 years.

A few clinical trials starting from 2008 showed that interferon is effective in normalizing blood counts, reducing the mutant JAK2 allele burden and, in some cases, reversing disease progression.

In 2010 EMA designated UDCA an "orphan medicine" status — a medicine used for the treatment of primary biliary cholangitis.

Tetrabenazine approval for the treatment of Huntington's chorea in 23 European countires till the end of 2011.

Nabilone is a synthetically produced substance belonging to a group of compounds known as cannabionoids, used for the treatment of CINV.

The PROUD-PV study is a phase III study to compare the efficacy and safety of the novel ropeginterferon α-2b versus hydroxyurea in both HU-naive and currently treated patients, diagnosed with Polycythemia vera.

The paper “Beta-blocker use in severe sepsis and septic shock: a systematic review.” by Sanfilippo F1, Santonocito C2, Morelli A3, Foex P4 describes the ability of beta blockade to modulate sepsis-induced alterations at the cardiovascular, metabolic, immunologic and coagulation levels.

Pitolisant, also known as tiprolisant, is a potent and selective inverse agonist of the histamine H3 receptor (Ki = 0.16 nM), and was approved for the treatment of narcolepsy in 2016. Pitolisant was developed by Jean-Charles Schwartz, Walter Schunack, and colleagues, after the former discovered the H3 receptor. It was the first H3 receptor inverse agonist to be tested in humans or introduced for clinical use.

Landiolol, a new ultra-high selective short acting beta 1 adrenoceptor blocker, developed by AOP in 2 forms (20 mg/2ml concentrate, 300 mg powder) is registered in several European countires for the treatment of non-compensatory sinus tachycardia and tachycardic supraventricular arrhythmias. The drug uses a new AOP Health-developed dosing algorithm to facilitate the administration and proves to be superior to Esmolol in its ability to reduce heart rate more than blood pressure.

The paper “Subcutaneous treprostinil for the treatment of severe non-operable chronic thromboembolic pulmonary hypertension (CTREPH): a double-blind, phase 3, randomised controlled trial" was published by R. Sadushi-Kolici R; Jansa P; Kopec G; Torbicki A; Skoro-Sajer N; Campean IA; Halank M; Simkova I; Karlocai K; Steringer-Mascherbauer R; Samarzija M; Salobir B; Klepetko W; Lindner J; Lang IM (Source: https://www.thelancet.com/journals/lanres/article/PIIS2213-2600(18)30367-9/fulltext). The authors conclude that sc. Treprostinil significantly improves the cardiovascular situation and performances figure of patients with CTREPH.

On Feb 15th EMA approves AOP Health’s BESREMi® as firt-line monotherapy in adults for the treatment of Polycytheamia vera (PV) without symptomatic splenomegaly.