News brief­ing: A sweep of CHMP OKs for Al­ny­lam, Kite, No­var­tis and oth­ers; Sanofi's big can­cer play gets OK from UK watch­dog

At this month’s meet­ing, the EMA’s Com­mit­tee for Med­i­c­i­nal Prod­ucts for Hu­man Use is­sued rec­om­men­da­tions of ap­proval for 10 drugs. They in­clude:

• Oxlu­mo (lumasir­an), Al­ny­lam’s RNAi can­di­date that snagged pri­or­i­ty re­view from the FDA back in May. In June, the biotech re­leased piv­otal da­ta which showed that 21 of 25 pa­tients with PH1 (pri­ma­ry hy­per­ox­aluria type 1), a rare liv­er con­di­tion, saw their ox­alate lev­els fall to near the nor­mal range af­ter be­ing treat­ed with lumasir­an, and just over half had num­bers in­side the nor­mal range. That was com­pared to 0% in the place­bo group, ac­cord­ing to Al­ny­lam.
• Kite’s Tecar­tus (KTE-X19), a CAR-T ther­a­py ap­proved by the FDA in Ju­ly to treat adult pa­tients with re­lapsed or re­frac­to­ry man­tle cell lym­phoma, a rare form of NHL. Ken Takeshi­ta, head of glob­al de­vel­op­ment at Gilead sub­sidiary Kite Ther­a­peu­tics, boast­ed that the drug’s da­ta were bet­ter than the com­pa­ny’s CAR-T drug Yescar­ta, which was ap­proved in 2017. A com­pa­ny spokesper­son said in Ju­ly that the drug will be sold for $373,000.
• No­var­tis’ Leqvio (in­clisir­an), the LDL-C drug that was for­mer­ly a Med­i­cines Com­pa­ny can­di­date. An Au­gust read-out from two Phase III tri­als showed con­sis­ten­cy in ef­fi­ca­cy and safe­ty among pa­tients with both hy­per­lipi­demia and ath­er­o­scle­rot­ic car­dio­vas­cu­lar dis­ease de­spite statin ther­a­py, ac­cord­ing to the com­pa­ny. Around 88% of pa­tients hit 50% re­duc­tion of LDL-C in at least one point dur­ing the 17-month study. Af­ter the 17-month mark, al­most two-thirds of pa­tients (66.4%) had a 50% re­duc­tion com­pared to 2.5% per­cent in the place­bo group.
• Or­chard Ther­a­peu­tics’ Lib­meldy for metachro­mat­ic leukody­s­tro­phy
• Janssen’s Rekam­bys for hu­man im­mun­od­e­fi­cien­cy virus type 1 (HIV-1)
• Vi­iV Health­care’s Vo­cabria for HIV-1
• Zo­genix’s Fin­tepla for the treat­ment of seizures as­so­ci­at­ed with Dravet syn­drome
• Aim­mune’s Pal­forzia for de­sen­si­tis­ing chil­dren and ado­les­cents to peanut al­ler­gy
• As­traZeneca’s Trix­eo Aeros­phere for the main­te­nance treat­ment of chron­ic ob­struc­tive pul­monary dis­ease
• My­lan’s lenalido­mide for the treat­ment of mul­ti­ple myelo­ma and fol­lic­u­lar lym­phoma

— Nicole De­Feud­is 

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER