Nusinersen: The Case for FDA Approval Now

Transcription

1 Nu: T C FDA Appv Nw

2 Tb C Nu F S... 3 Cmpv C w Ey Appv by FDA... 8 Nu C T D Ru Summy... 14

3 My Bz: K My: Fm A SMA Tm T FAST Mvm W p gup p, vu w SMA, upp gz 2015 bv by I Pmu y w v m Sp Muu Apy (SMA) y v v. T m Nu. W upp u SMA, bu g mpg y Nu. W v p w m 124,000 gu Dmb 19, 2015, g Cg FDA xp Nu ppv. W upp mup vgg m SMA u uwy uv mp u w. Hwv, Nu mp gu xpm p Nu uu p. Bu y y yg, w up FDA p Nu SMA, #1 g k. W Sp Muu Apy (SMA)? SMA g w vu mg pmy g pb g p Suvv M Nu (SMN). T pmy g SMN1, um, bk up g, SMN2, bu g pv m pug uy u SMN p. SMN p uvv v p pb umuu gw u. T k SMN u k mu pgvy py, g mu. T mu p by mv, ww, b. T mu SMN p pu m vy SMA. I m v m SMA, Typ 1, 50% bb xp v u by 90% xp v u by. SMA #1 g k u 2 y. F w uvv w SMA Typ 1, 2, 3, 4, v py u y mu. M myg bk up g w u upp SMN pv m u u u. A m, NO EXISTING TREATMENTS SMA.

4 My Bz: K My: Wy u Nu b vb y? T FDA g Nu p ug g bu wu uy p. Op ug g pv v ug vpm, u x, u wv, gby mk xuvy. Nu w g F Tk Dg pv ug vpm w ug bg pv m p. I 2011, N Iu H N Iu Nug D Sk SMA #1 m m 600 ug w v g w Nu. Nu ug I Pmu (my I Pm) vp b w D. A R. K C Spg Hb Lby. I g Nu pp w SMA. SMA v m u k m yug y g. SMA u m mu g pb pug p m u u. Nu g pu p by myg pg y g, by mpg uyg g. I Pmu up m g pgm Nu w w m v m v b gg pgvy g vg g w kg ug. Ag y pm My 15, 2015, Nu u ub mu mu u. Mu mu u 9 m vu m m mbuy p upp mb mbuy p mug p uy. Nu u w v gg.

5 My Bz: K My: "B w wu xp v v [mg vy p ] by 6 10 m, 10 u. W w'v xy m w xp Ty vg g, vg m mpy u. Fv Typ 1 p w b u. W w p [ uy] w g y. W v m w'." Sy Ck, M.D., PD., CEO I Pmu p: m g g v g ug I Pmu y p mup pp, mg y w y k FDA ppv g p v b. Hwv, wu xu mjy p ppu, ug u w v b vg w g. I m p v vg, k ppv xug m m ppuy v, ug y uu, xpb gup pp, wy upb. A pp w SMA v m mg g mp u. Obvuy, g w vy b g, wg, pg, bu b by py Nu ppv. Wv I pp ppv Nu, pmu y k ppv mzg m ALL p, ug w v b gg g. Bu, y, my m w b Nu ppv, w bv I u Nu Exp A (Cmp U) w. Epy g I xp p w w v b vg w g, p ppu b xp w y g ppv. Ev ppv p w bgu y, g m ubm ppwk wu p wu u z v. Cuy, I Exp A Pgm xp ppy. Aby NY, O. 20, 2015, (GLOBE NEWSWIRE) Ag w mk p pub by Tpy Mk, R Sp Muu Apy Mk: Pp Am, Sz, Gw, T, F, , Nu xp g vu USD 0.47 b by Ag p mk p, u Skg Ap Sk Mk Nw, I Pmu Mv C Dub Su, xp vu u x USD 4 b. Nm & Cmpy y, D. C M, Ju 23, 2015, "I y p up m gg u w Nu. W m mp w y u. Impy, Nu b uvv mpvm w mgu u mpvm. P 3 u w uwy bu w u b y m by Nu y x w y ppv y p p."

6 My Bz: K My: Cb, CA, Ju 22, 2015 (PRNww) I up pv by I Pmu, D. Dy D Vv, P Nugy P, Cumb Uvy M C Nu vg, "T u u w u yp II yp III SMA ypy xp mu u vp wy uy v m... u m p Hmmm M Fu S Exp [wg m w Nu] p g pu m u u uuu." C. Fk B, P.D., v p I Pmu mm, "I u ug mup mum mu m u g, w bv ugg u w w u m u p b P 2 uy. Tk g, ugg ISIS SMN Rx [ m Nu] u pv b p w SMA by g pg. Ag u m.gv, p b, p 3, SHINE, xp b mp u FAST Mvm I W vy ggg ky U.S. S Mmb Cg, ug S Cmm H, Eu, Lb & P (HELP) p w k FDA w Nu y. W u k FDA xp ppv Nu. W up Cg FDA xp Nu xp y buu b u gb p g Bg p vm. W w g Nu m z ug p u vpm. Y bug m bgy. W bv Nu v y v v y. W u upp gg SMA my g my y u. U, Nu pv u g uvv u.

7 My Bz: K My: C m: Bg I I Pmu, I. 225 By S 2855 Gz Cu Cmbg, MA Cb, CA Gvm R p: 601 Pyv Av., N.W. Su 720 Wg, DC U.S. S Cmm H, Eu, Lb & P U.S. F Dug Am p: T F (855) , (301) S Dk O Bug ug@..gv Wg, DC Hum Dug Im: Dv Dug Im (CDER) O Cmmu Fbk Fm Nw Hmp Avu H Bug, 4 F Sv Spg, MD 20993

8 Cmpv C Appv by F Dug Am Iu T F Dug Am (FDA) p mv xby g p uy v m vb w p ppu m -xpy. Nu ug py v v p w Sp Muu Apy (SMA). SMA xmp w xby mu b u. SMA umb g k, 1 6,000 v b p y uvv. I Quum Ev Ev FDA' Appv Op Dug, Fk Swk 1, Cm B, N Ogz R D FDA puby xp vy ppyg xby w p. F xmp, my U S S Ju 23, 2010, D. J Gm, FDA C S Dpuy Cmm S Pub H, yg FDA E R Ng D FDA uy mm ppyg qu xby vpm vw pu, w ug mp pby u pu v gy vub ppu. S bw xmp ug ppv g w FDA xby vy b ug vpm bug uu m. My 357 ppv p ug w m umb p, vg m FDA mmm p. T pb w b pp w p m v. T b FDA ppv ug wg w gu uy u by I Pmu pp w Bg I Nu. W FDA by ppv ug, wg, y p, mm p ppu, wu qug (pb) m uy, I w p-b, p-p 3, u m p gg p 3 uy m, ug 1:2 pb gup, u p 3 v p 4 uy m p. W y vg wg m, u p, FDA mpy mv xby g py, m w uy y v v ppv m vb, jub FDA uz xby SMA.

9 Tm U A Appv - w FDA ppv pu u -g b v pu um by ky p b. Bkug Tpy Dg - FDA xp ug vpm, w w FDA u S 902 Juy 9, 2012 F Dug Am Sy Iv A, g py vw ug pmy py my ub m vg v xg p p w u -g. Op Dug Dg - pv p u ug bg w v m, g, pv w 200,000 pp U.S., m 200,000 p bu xp v vpg mkg m ug. Op Dug Dg pv v u x, u wv, gby xuvy ug vpm ug. Py Rvw Su - xp vw g by FDA vp m g. Appv Dug Kum 2 : Appv Dmb 8, 2015 m p w kw ym p (LAL) y, kw Wm y g (CESD). Sympm ypy p bw 2 4 m, p y xp v by y. Tm pv v vu u wky w py pgv LAL y pg x m, vy wk p. T C Dug Evu R vu y y Kum p-b, y w py pgv Wm ub-b, pb- 66 p u p w CESD. I w Wm, x (67 p) w Kum w v 12 m g, w 21 gup uvv. I CESD p, w y g mpvm LDL v - pm w Kum vu pb 20 wk m. 1 p Sm p ppu Nu y u pb uy Vy p yp m uy Appv ALL p S FDA P Aum ppv Kum. Xu 3 : Appv Spmb 14, 2015 p w y u, mb, w b p ppxmy 20 p ww. T y

10 v Xu w vu g m, 6-wk, p-b u p w y u, gg g m 3 19 y g, 6-m x p. T uy g p p-p mg pm ug p. A b 6-wk 6-m m, Xu m u by mg pm u p. T y v u pm py w u upp by p m pub u. 1 p N pb m Sm p ppu Appv S FDA P Aum gg Xu' ppv. Tg 4 : Lug m ppv Nvmb 2015 p FDA A A Pgm. T y y Tg w m w mu, g-m u vvg 411 p w v EGFR T790M mu-pv NSCLC w w m w EGFR-bkg m. I w u, 57% p uy 61% p uy xp mp p u um z (kw bjv p ). 1 p N pb m Appv S FDA P Aum gg Tg' ppv. Pbb 5 : B m g Bkug Tpy g Ap O Fbuy 3, 2015, U.S. F Dug Am g ppv Pbb (IBRANCE, Pz, I.) u mb w z m pmpu wm w g p (ER)-pv, um pm gw p 2 (HER2)- gv v b -b py m. Pbb w mz, mu, p-b 165 wm, my v pbb pu z z. Ov p p w mub (vg m) w g pbb pu z mp z m, ug w ppv, v ug my p (g 10%) xp mup, m v xbg u v, u pumy mbm. 1 p N pb m Sm p ppu Av p Appv S FDA P Aum gg Pbb' ppv.

11 A 6 : Appv Dmb 2015 m ALK-pv ug. T y y A w u w g-m p w m w w g by m w v ug. Suy pp v A w y mu ug ug um. I uy, 38% pp xp p kg um, vg 7.5 m. I uy, 44 p pp xp p kg um, g vg 11.2 m. T xm A vu b m, mm u ppu. 61% pp w w mub b m xp mp p u b um, g vg 9.1 m. A wu, m w b -g. T FDA g A pp bkug py g, py vw u, p ug g. 2 p N pb m Av p Appv S FDA P Aum gg A' ppv. Czym 7 : Ezym-pm py u Gu', ppv w m p m T ppv Czym w b P 3 uy mpg mb v C (ppv 1991), w vv 30 p. T p, w w p vy bw w m, v p ug v p x m. T uy u w p Czym- m by g m w v C (16% vu 40%). N pb m Sm p ppu Appv S P Aum gg ppv Czym. Myzym 8 : Ezym py ppv Ap 2006 um zym y w Pmp. Appv Myzym w b p, w mb 39 p w Pmp. Suy 1, 18 p w u bg mu g b (u y). Suy 2, 21 p (3 m-3.5 y g) w y bg mu puy by mg v uy. 2 p N pb m (u y )

12 Sm p ppu Appv S P Aum ppv Myzym. Cu T FDA pb pg pub by ug y, y uy um... ug... vg pub by pg p v mk m m v,, m b. 9 SMA p m vu FDA u y ug w y b v, bu. FAST pp w vb FDA ju k SMA. T p vb p m SMA xpy vg vg w w y m b ppv. T bg, y m-v u jugm. T u p Nu by I Pmu v b g bkg-- upg v xp u. T, v b ug- u v v p w Nu. T u y m ug w ug y u pb gup u p ug p v pb y uy, bu u. Ev, pb m bg Ju 2014, w u b m u, py w g u y mu-uy. T FAST Mvm kg ky U.S. S Mmb Cg, ug S Cmm H, Eu, Lb & P (HELP) p w k FDA w Nu y. W u k FDA xp ppv Nu, w up Cg FDA xp Nu xp y buu b u gb p g I-Bg p vm. W bv Nu, v, y v v y.

13 R 1 Swk, Fk J., M.S., M.P.H., J.D. Quum Ev Ev FDA' Appv Op Dug: Cgug FDA' Fxby Rgug Tp P w R D. Fb <p:pygu.gwp-up201202norduy--fda-ppv--p-ug.p> 2 U.S. F Dug Am. FDA Nw R: FDA Appv F Dug T R Ezym D P Au P. D 8, <p: NwEvNwmPAumum m> 3 U.S. F Dug Am. FDA Nw R: FDA Appv Nw Op Dug T R Aum Rv D. Sp 4, <p: NwmPAumum m> 4 U.S. F Dug Am. FDA Nw R: FDA Appv Nw P T C P w N-Sm C Lug C. Nv 13, <p: NwmPAumum m> 5 U.S. F Dug Am. Pbb. Fb 4, <p: ImODugAppvDugum m> 6 U.S. F Dug Am. FDA Nw R: FDA Appv Nw O Tpy T ALK-Pv Lug C. D 11, p: PAumum m> 7 TPmL. Czym Appv USA. Ju 6, <p: zym-ppv--u> 8 CW. Myzym (Agu A)... <p: 9 U.S. F Dug Am. Abu FDA: W W D. D 7, <p: gvabufdawwd>

14 Nu p u Ap A p p v b yi g Nu b w v w : p : b. y 1 Px J I Wk p : j p m g. m m g. m b k b k _ w. p p? w b =2 T u ysmaty p1 p m v m w m DECLI NE CHOP p p y I w Nu, w m g1 7p CHOP w 1 5m 5 3 % 4 0m p CHOP,4 0 w m 8 0 % v u v v w Nu v 1 8 % v u v v u Augu u w m g CHOP, m 1 7p g I SI SSMNRx b w w y N ug SAE ;m j ysaew p y Av Ev ( SAE )m ym m v y Np D L m gtx p N ug g u g x m N y g g CSF y b N g yp w p j