EMA gives thumbs up to key drugs from No­var­tis, Alex­ion, Ver­tex and more

→ The EMA’s Com­mit­tee for Med­i­c­i­nal Prod­ucts for Hu­man Use has adopt­ed pos­i­tive opin­ions for a slate of drugs, is­su­ing rec­om­men­da­tion ap­provals that the EU will very like­ly fol­low. They in­clude:

• En­erzair (QVM149), one of the key drugs that No­var­tis CEO Vas Narasimhan has tapped as a po­ten­tial block­buster. The asth­ma treat­ment com­pris­es a long-act­ing be­ta-ag­o­nist, or LA­BA, called in­da­caterol ac­etate, a long-act­ing mus­carinic an­tag­o­nist in gly­copy­rro­ni­um bro­mide, and the cor­ti­cos­teroid mometa­sone furoate. The prod­uct would be ad­min­is­tered once-dai­ly through an in­haler.
• Alex­ion’s Ul­tomiris for atyp­i­cal he­molyt­ic ure­mic syn­drome (aHUS) — for both com­ple­ment in­hibitor treat­ment-naïve pa­tients and any­one who’s been re­spond­ing to Soliris. If ap­proved, it would be the first and on­ly long-act­ing C5 in­hibitor for the ul­tra-rare but se­ri­ous in­di­ca­tion in Eu­rope, R&D chief John Orloff not­ed. In two on­go­ing sin­gle-arm, open-la­bel stud­ies, Ul­tomiris helped adult pa­tients and chil­dren nor­mal­ize cer­tain blood pa­ra­me­ters and im­prove kid­ney func­tion at an in­ter­im analy­sis.
• A la­bel ex­pan­sion for Ver­tex’s CF ther­a­py Ka­ly­de­co, which has pre­vi­ous­ly been the cen­ter of heat­ed pric­ing de­bates with pay­ers. An OK here would cov­er chil­dren and ado­les­cents be­tween 6 months and 18 years old who have the R117H mu­ta­tion on the CFTR gene. “To­day’s an­nounce­ment is im­por­tant for young peo­ple with CF, as ear­ly in­ter­ven­tion and treat­ment of this dev­as­tat­ing and pro­gres­sive dis­ease is key to keep­ing pa­tients health­i­er longer,” Car­men Boz­ic, who’s been pro­mot­ed to the chief med­ical of­fi­cer role as Resh­ma Ke­wal­ra­mani takes the helm, in a state­ment.
• CHMP has rec­om­mend­ed for ap­proval a sub­cu­ta­neous form of J&J’s mul­ti­ple myelo­ma drug Darza­lex. The for­mu­la­tion us­es Halozyme’s tech­nol­o­gy to de­liv­er the drug in five min­utes as op­posed to over sev­er­al hours by IV in­fu­sion, the biotech said.

→ Achieve Life Sci­ences, a Cana­di­an biotech de­vel­op­ing a drug for nico­tine ces­sa­tion, has ex­e­cut­ed a $1.9 mil­lion pri­vate place­ment for its stock, at $0.33 per share. The com­pa­ny has had mixed suc­cess in late-stage clin­i­cal tri­als, but con­cerns about patent abil­i­ty of its prod­uct — which has long been avail­able in parts of Eu­rope — and com­ing gener­ics for Chan­tix have killed its stock price.

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