Or­chard halts dos­ing of EMA-ap­proved Strimvelis af­ter pa­tient de­vel­ops leukemia

Or­chard Ther­a­peu­tics is pulling its EMA-ap­proved gene ther­a­py Strimvelis off the shelves while it in­ves­ti­gates a po­ten­tial link be­tween the drug and a case of lym­phoid T-cell leukemia.

The biotech $OR­TX said it was no­ti­fied of the ad­verse event a few days ago — just months af­ter it slashed 25% of its work­force in an at­tempt to stray away from rare dis­eases like “bub­ble boy syn­drome,” the one Strimvelis is ap­proved for. The pa­tient was treat­ed un­der a com­pas­sion­ate use pro­gram in 2016, and is now be­ing treat­ed for leukemia at a spe­cial­ty cen­ter. Pre­lim­i­nary find­ings sug­gest the di­ag­no­sis could be linked to an in­ser­tion­al event re­lat­ed to the treat­ment, Or­chard an­nounced.

“No ad­di­tion­al pa­tients will be treat­ed with Strimvelis be­fore the in­ves­ti­ga­tion is com­plete,” the com­pa­ny said in an emailed state­ment. When asked how long the probe might take, the biotech re­spond­ed that an in­ves­ti­ga­tion is un­der­way, and that more in­for­ma­tion will be avail­able once it’s com­plete.

“Or­chard has no­ti­fied EMA of this event, and we re­main in col­lab­o­ra­tive and on­go­ing di­a­logue with the agency,” spokesper­son Chris­tine Har­ri­son said.

Strimvelis was ap­proved in 2016 for pa­tients with se­vere com­bined im­mun­od­e­fi­cien­cy due to adeno­sine deam­i­nase de­fi­cien­cy (ADA-SCID). Af­ter at­tempt­ing to sell off its gene ther­a­py unit for months, Glax­o­SmithK­line trad­ed it to Or­chard in 2018 along with oth­er gene ther­a­py pro­grams for a near­ly 20% stake in the com­pa­ny.

The British drug­mak­er — and lat­er, Or­chard — strug­gled to find cus­tomers among the rare ADA-SCID pa­tient pop­u­la­tion. To date, on­ly 16 peo­ple have been treat­ed with the drug since it was ap­proved.

Back in May, the com­pa­ny re­or­ga­nized to fo­cus on more preva­lent ill­ness­es. It cut 25% of em­ploy­ees, halt­ed the con­struc­tion of a Fre­mont man­u­fac­tur­ing fa­cil­i­ty, shut down a Cal­i­for­nia R&D site, and di­vest­ed two pro­grams (an­oth­er ACA-SCID gene ther­a­py can­di­date and a trans­fu­sion-de­pen­dent be­ta-tha­lassemia can­di­date). When an­nounc­ing the shake­up, Or­chard said it would pri­or­i­tize pro­grams for metachro­mat­ic leukody­s­tro­phy (OTL-200), Wiskott-Aldrich syn­drome (OTL-103), Mu­copolysac­cha­ri­do­sis type I (OTL-203) and Mu­copolysac­cha­ri­do­sis type II­IA, al­so known as San­fil­ip­po syn­drome type A (OTL-201).

Or­chard said that leukemia re­sult­ing from in­ser­tion­al onco­ge­n­e­sis is a known risk of gam­maretro­vi­ral vec­tor-based gene ther­a­py, as de­scribed on the Strimvelis warn­ing la­bel.

The EMA’s prod­uct sum­ma­ry reads:

No cas­es of leukaemia or myelodys­pla­sia have been re­port­ed fol­low­ing treat­ment with Strimvelis. How­ev­er,vec­tor in­ser­tions in­to chro­mo­so­mal re­gions pre­vi­ous­ly as­so­ci­at­ed with leukaemia in com­pa­ra­ble tri­als of­gene ther­a­py in Wiskott Aldrich Syn­drome, X-SCID and Chron­ic Gran­u­lo­ma­tous Dis­ease have been­doc­u­ment­ed. Retro­vi­ral in­ser­tion sites (RIS) have been de­tect­ed ad­ja­cent to or with­in CC­ND2 and LMO2and there is a po­ten­tial risk of leukaemic trans­for­ma­tion fol­low­ing treat­ment with Strimvelis. It is­rec­om­mend­ed that pa­tients be mon­i­tored long term with at least an­nu­al vis­its for the first eleven years andthen at 13 and 15 years post treat­ment with Strimvelis, to in­clude a com­plete blood count with dif­fer­en­tial,bio­chem­istry and thy­roid stim­u­lat­ing hor­mone.

Or­chard’s stock has been on a steady de­cline since Jan­u­ary, when shares reached a high of $15.71 apiece. They closed Fri­day at $4.04 per share.

Strimvelis’ fu­ture re­mains un­clear. “The in­ves­ti­ga­tion is on­go­ing and the com­pa­ny will de­ter­mine the fu­ture of Strimvelis fol­low­ing dis­cus­sions with rel­e­vant stake­hold­ers and will pro­vide fur­ther up­dates as ap­pro­pri­ate,” Or­chard an­nounced.

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