BWD brought this up earlier this week (Arikayce for NTM):
Relating to the designation of "Amikacin sulfate" as an orphan medicinal product under
Regulation (EC) No 141/2000 of the European Parliament and of the Council
THE EUROPEAN COMMISSION,
Having regard to the Treaty on the Functioning of the European Union,
Having regard to Regulation (EC) No 141/2000 of the European Parliament and of the
Council of 16 December 1999 on orphan medicinal products1, and in particular the first
sentence of Article 5(8) thereof,
Having regard to the application submitted by Insmed Limited on 28 November 2013
under Article 5(1) of Regulation (EC) No 141/2000,
Having regard to the favourable opinion of the European Medicines Agency, drawn up
on 6 February 2014 by the Committee for Orphan Medicinal Products and received by
the Commission on 27 February 2014,
(1) The application submitted by Insmed Limited concerning the medicinal product
“Amikacin sulfate” was validated on 13 December 2013 under Article 5(4) of
Regulation (EC) No 141/2000.
(2) "Amikacin sulfate" meets the designation criteria referred to in Article 3(1) of
Regulation (EC) No 141/2000.
(3) The application should therefore be accepted,
HAS ADOPTED THIS DECISION:
The medicinal product “Amikacin sulfate” is designated as an orphan medicinal product
for the indication: Treatment of nontuberculous mycobacterial lung disease. It shall be
entered in the Community Register of Orphan Medicinal Products under number
The European Medicines Agency shall make available to all interested parties the
opinion of the Committee on Orphan Medicinal Products referred to in this Decision.
Inhalation amikacin sulfate solution (aka BAY41-6551) for Gram negative pneumonia. You should compare the studies. The world of antibiotic therapy clinical studies is not stationery.
Arikace will do much better against naïve patients that have never been treated with antibiotics following confirmed diagnosis of NTM. But that is in the future.
Arikace has a proven track-record for safety and efficacy in numerous animal models (mice, rats AND dogs) and multiple clinical trial trials already (phase 1, 2 and 3) for CF, bronchiectasis and NTM.
Insmed has already orphan status in EU and US for CF and NTM, AND the second highest "accelerated" status at the FDA for approval fast-track under the GAIN act (including priority review: review on-the-fly of whatever documentation is completed as/when it is completed - Insmed is already IN the process of filing - they are just not shouting about it).
It is highly likely that the FDA will grant breakthrough therapy status any day between now and mid May and even if it doesn't, marketing authorization is still likely for both CF and NTM in both EU and US by end of 2015.
So... sell/hold/whatever... makes NO difference to what is already on-going and on-track.
• culture conversions in 25% of RECALCITRANT NTM patients
• sustained efficacy for MONTHS in the case of CF/Pa
Of course Arikace will be even more impressive for naive patients; it will be the first line of treatment for any NTM diagnosis anyway from 2015/16 in Europe, North American and become globally dominant from 2018 for at least 10~12+ years
 "The world of antibiotic therapy clinical studies is not stationery". It is regarding NTM. Go ahead: see what other competitive studies you can find for new treatments for NTM...
 I suggest you surf the FDA website and find Novartis' own presentation in support of TIP (podhaler): 1.78Mb pdf file of 142 slides
Btw - if this means Insmed is now protected for a given period from competition in NTM it's massive news.
That would prevent competition both from amikacin injection in the treatment of NTM - and from any other inhaled therapy for NTM which incorporates amikacin.
Not good news for Aradigm if that's true. And isn't there another inhaled amikacin product currently in Phase III?
The EMA had already designated NTM as an Orphan condition.
Insmed apparently asked the EMA to designate Arikace as an Orphan medicinal product for NTM.
The EMA designated Amikacin Sulfate as an Orphan medicinal product for NTM - presumably deeming the liposomal delivery vehicle inert.
Very handy imo - as I seem to recall reading (in a patent?) that Insmed intended the product to be partly comprised of unencapsulated amikacin.
1. February 10 2014 -
Insmed Incorporated ... today announced that the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA) has issued a positive opinion on the Company's application for orphan designation for ARIKACE®, the Company's liposomal amikacin for inhalation, for the treatment of lung disease caused by nontuberculous mycobacteria (NTM).
2. March 6 2014 (10-K) -
In December 2010, we received Positive Opinion of the Pediatric Committee of the EMA on the agreement of our PIP, on the granting of a deferral, and on the granting of a waiver for amikacin (sulfate) nebulizer suspension for inhalation use, in the treatment of Pseudomonas lung infection/colonization in CF patients in accordance with relevant European regulations. Our PIP is subject to modifications from time to time.
ARIKAYCE has been granted the following orphan drug designations ...
European Union (EU): NTM lung infections and Pseudomonas lung infections in CF patients.
3. April 8 2014 -
Having regard to the application submitted by Insmed Limited on 28 November 2013 under Article 5(1) of Regulation (EC) No 141/2000,
Having regard to the favourable opinion of the European Medicines Agency, drawn upon 6 February 2014 by the Committee for Orphan Medicinal Products and received by the Commission on 27 February 2014 ...
The medicinal product "Amikacin sulfate" is designated as an orphan medicinal product for the indication: Treatment of nontuberculous mycobacterial lung disease.
So, can we now start selling Aryikace in Europe? And, it is my understanding that most of the world accepts EMA approvals and, therefore, we can sell globally. Only the FDA at this point is denying the sale of Arykace and only Americans are denied access to this lifesaving product.
FUD then what was the EC referring to on April 8th? I never saw/read Arikayce/Arikace in their decision. So, I thought it was "inherent" yet as you suggest, when I looked up amikacin sulfate/Insmed I get:
"The FDA has indicated that it considers ARIKAYCE a NME for application and review purposes even though the agency has previously approved drugs with the active ingredient, amikacin sulfate. FDA characterizes some drugs as NMEs for administrative purposes, even if they contain an active moiety (the molecule or ion responsible for the action of the drug substance) that is closely related to active moieties in products that have previously been approved by FDA. Amikacin sulfate is an FDA-approved antibiotic with proven efficacy in the treatment of a broad range of gram-negative infections, including Pseudomonas and NTM. ARIKAYCE is in the aminoglycoside class of antibiotics."