1. INSM files in Europe for EMEA approval of CF/Pa at end of year after Chiltren International report is released on trial results compared to TOBI. CHMP moves to approve quickly (probably early 2014) after cGMP manufacturing is established in Europe. Minimally Arikace fits into a rotation of inhalation antibiotics that will start to reverse the incidence of resistant Pa strains.
2. NTM results show significant numbers of eradicated NTM infections among patients and this level of efficacy occurs in less than 84 days of treatment. Compassionate use and NDA are filed in quick succession. cGMP is probably already near validation in US.
3. Based upon 1 above and cGMP in US, an NDA is filed in the US for CF/Pa probably in early 2014.
4. Based upon compellation of control patient results for liposome, an NDA masterfile is submitted to FDA and EMEA to permit expanded clinical trials with other inhalational liposome formulations. Other formulations would include small molecules, large molecules, biologics and gene altering sequences. Also probably in 2014.
These four steps take inhalation preparations forward to a new position in therapeutic medicine and are within a reasonable time for Longs to hold this investment. They are also probably attainable within one year. Good news for Longs.