Astrazeneca Share News (AZN)

(Sharecast News) - AstraZeneca announced on Wednesday morning that the US Food and Drug Administration (FDA) has granted 'Orphan Drug Designation' to 'Fasenra' (benralizumab), for the treatment of eosinophilic oesophagitis (EoE).
The FTSE 100 pharmaceuticals giant also released positive results from the phase 3 'ETHOS' trial for triple-combination therapy 'Breztri Aerosphere', formerly 'PT010', in patients with moderate-to-very severe chronic obstructive pulmonary disease (COPD).

On the designation of Fasenra as an orphan drug, AstraZeneca said EoE is a "rare, chronic, inflammatory disease" that occured when eosinophils - a type of white blood cell - accumulated in the oesophagus, causing injury and inflammation.

It said the FDA grants Orphan Drug Designation status to medicines and potential new medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affected fewer than 200,000 people in the United States.

"Eosinophilic oesophagitis is an allergic inflammatory disease that can affect children and adults, and typically patients experience a range of debilitating symptoms including severe pain and difficulty swallowing food," said AstraZeneca's executive vice president of biopharmaceuticals research and development, Mene Pangalos.

"Currently there are no FDA-approved treatments for eosinophilic oesophagitis.

"Because Fasenra depletes eosinophils in blood and tissue, it could become a potential new medicine to treat patients with this rare disease."

The firm explained that Fasenra was its first respiratory biologic medicine, and was currently approved as an add-on maintenance treatment for severe eosinophilic asthma in the US, European Union, Japan and other countries.

In November last year, the FDA granted Orphan Drug Designation for Fasenra for the treatment of eosinophilic granulomatosis with polyangiitis (EGPA), and also granted it for the treatment of hypereosinophilic syndrome (HES) in February.

On the phase 3 results for Breztri Aerosphere, AstraZeneca said that at the standard budesonide dose, the treatment - which contained budesonide, glycopyrronium and formoterol fumarate at 320 mcg, 14.4 mcg and 9.6 mcg respectively, demonstrated a "statistically-significant reduction" in the rate of moderate or severe exacerbations compared with dual-combination therapies Bevespi Aerosphere and 'PT009', which is budesonide and formoterol fumarate at 320 and 9.6mcg.

At half of the budesonide dose, Breztri Aerosphere also reportedly demonstrated a statistically-significant reduction in the rate of moderate or severe exacerbations, compared with Bevespi Aerosphere and PT009.

The company said the dual-combination therapies used as comparators in the trial represented recommended therapeutic classes for the treatment of COPD.

"Exacerbations are devastating events for patients and can lead to a permanent loss of lung function," Mene Pangalos explained.

"The phase 3 ETHOS trial builds on the phase 3 KRONOS data, which together show Breztri Aerosphere's ability to reduce exacerbation risk in a broad range of patients with COPD, irrespective of whether they have had an exacerbation in the previous 12 months.

"We look forward to sharing these results with health authorities as soon as possible."

Klaus Rabe, professor of pulmonary medicine at the University of Kiel, director of the department of pneumology at Clinic Grosshansdorf in Germany, and lead investigator of the ETHOS trial, added that the phase 3 ETHOS trial results were "exciting", and demonstrated that Breztri Aerosphere "significantly reduced" the rate of exacerbations.

"This is also the first time we have seen the benefit of fixed-dose triple-combination therapy at two inhaled corticosteroid doses, which could transform treatment practice by allowing physicians to select the optimal dose for individual patients."

AstraZeneca said the safety and tolerability of Breztri Aerosphere was consistent with the known profiles of the dual comparators.

In the trial, all combination therapies were administered in a pressurised metered-dose inhaler using 'Aerosphere' delivery technology.

The ETHOS trial results would be presented at an upcoming medical meeting.

Breztri Aerosphere was approved in Japan, and was under regulatory review in China, where it had been granted 'Priority Review' status by the National Medical Products Administration.

It was also under regulatory review in the US and the EU.