Hello Everyone, Congratulations to all Pharmaxis holders on the...

Hello Everyone,

Congratulations to all Pharmaxis holders on the Bronchitol obtaining US FDA approval (Screenshot below),


Bronchitol for cystic fibrosis
Bronchitol, an inhaled dry powder for the treatment of cystic fibrosis, has been the subject of three large scale global clinical trials conducted by Pharmaxis.

• In the EU, Pharmaxis has appointed Chiesi as its exclusive distributor for the markets of the UK, Ireland, Italy, Germany, Norway, Sweden, Finland, Denmark, Cyprus and Greece. During the year the Chiesi territory was expanded to include Spain.

• In Russia, Bronchitol was approved for both adult and paediatric CF patients in 2016 and has been reimbursed nationally since January 2019.

• During the year the Company received approval for Bronchitol in Brazil. The next step is filing a reimbursement application.

• Pharmaxis has partnered its work on Bronchitol for the United States with Chiesi Group (Chiesi), a global pharmaceutical company headquartered in Parma, Italy. Chiesi USA, the American affiliate of Chiesi Group is responsible for completing and filing the updated Bronchitol NDA with the FDA. Based on the international Phase 3 trial of Bronchitol in adults with cystic fibrosis that reported in June 2017, Chiesi filed a resubmission of the Bronchitol New Drug Application (NDA) to the US Food and Drug Administration (FDA) in December 2018. Following a positive recommendation from a Pulmonary-Allergy Drugs Advisory Committee meeting convened by the FDA on 8 May 2019, Chiesi received a complete response letter from the FDA in June 2019 detailing the remaining matters to be addressed before Bronchitol can be approved for adult cystic fibrosis patients in the United States. The main requirement included in the FDA complete response letter was that Chiesi revise the product packaging and user instructions; and then conduct a human factor study (HFS) demonstrating that the revised user components enable healthcare professionals to properly administer the mannitol tolerance test – an initial test to ensure patients hypersensitive to mannitol are not prescribed Bronchitol. Company received US FDA on Friday, 31 October 2020 now

Cystic Fibroris addressable market in US
Cystic Fibrosis (C”) is a serious progressive genetic disease that causes persistent lung infections and cumulative, progressive and irreversible lung damage that severely limits the quality and length of life of those living with the condition. According to the US based Cystic Fibrosis
Foundation, more than 70,000 people worldwide suffer from CF with approximately 1,000 new cases of CF being diagnosed each year in the US alone. In the USA it is estimated that 1 in 25 Caucasian people are carriers of one mutation of the CF gene1.

Mannitol business unit (Bronchitol and Aridol) nearing breakeven revenues. PXS will be given $10mil, given their agreement with a major biotech upon FDA approval.
In my opinion, revenue from Cystic fibrosis related drug will be huge, Given Vertex Pharmaceutical Full-year 2018 CF product revenues were $3.04 billion.
https://investors.vrtx.com/node/25791/pdf

Other opportunities in the pipeline



Exciting times for the Company. Good luck to all.

All in my opinion please do your own research.