London, United Kingdom (Press Release) – GlaxoSmithKline plc (LSE/NYSE: GSK) to­day an­nounced that it has re­ceived Break­through Therapy Desig­na­tion from the U.S. Food and Drug Admin­istra­tion (FDA) for GSK2857916 mono­­therapy in patients with mul­ti­ple myeloma who have failed at least three prior lines of ther­apy, in­­clud­ing an anti-CD38 anti­body and are re­frac­tory to a pro­te­a­some in­hib­i­tor and an immuno­modulatory agent. In Octo­ber, the Euro­pean Medicines Agency (EMA) granted PRIME desig­na­tion to GSK2857916 for the treat­ment of re­lapsed and re­frac­tory mul­ti­ple myeloma patients whose prior ther­apy in­cluded a pro­te­a­some in­hib­i­tor, an immuno­modulatory agent and an anti-CD38 anti­body. GSK2857916 is an anti B-cell maturation agent (BCMA) mono­clonal anti­body-drug con­ju­gate.

GSK2857916 has also re­ceived orphan drug desig­na­tion from the EMA and FDA for mul­ti­ple myeloma.

The PRIME and Break­through Therapy Desig­na­tions are based on re­­sults from a phase 1 open-label, dose escalation and ex­pan­sion study in patients with re­lapsed/​refractory mul­ti­ple myeloma, irrespective of BCMA ex­pres­sion. Data from this on­go­ing trial will be pre­sented on 11th De­cem­ber in an oral pre­sen­ta­tion at the 59th annual meeting of the American Society of He­ma­tol­ogy meeting in Atlanta.

Axel Hoos, SVP Oncology R&D, GSK said "Oncology R&D at GSK is focussed on devel­op­ing med­i­cines with trans­formational poten­tial for patients and we are pleased that our inves­ti­ga­tional anti­body-drug con­ju­gate is the first BCMA targeting agent to re­ceive Break­through Therapy and PRIME desig­na­tion. GSK plans to rapidly ad­vance clin­i­cal trials with this promising ther­apy, alone and in com­bi­na­tion with other ther­a­pies, to fur­ther in­ves­ti­gate how GSK2857916 could ben­e­fit patients with mul­ti­ple myeloma. The mono­therapy data that we have seen for GSK2857916 sup­port its trans­formational poten­tial and we look for­ward to work­ing with regulators as we progress the devel­op­ment pro­gramme."

Notes to editors

Breakthrough Therapy Desig­na­tion is de­signed to expedite the devel­op­ment and re­view of drugs that are in­tended to treat a serious con­di­tion and pre­lim­i­nary clin­i­cal evi­dence in­di­cates that the drug may dem­onstrate sub­stan­tial im­prove­ment over avail­able ther­apy on a clin­i­cally sig­nif­i­cant end­point(s). Drugs that re­ceive break­­through ther­apy desig­na­tion are eli­gible for all features of FDA's Fast Track Programme.[i]

PRIME desig­na­tion is offered by the Euro­pean Medicines Agency (EMA) to en­hance sup­port for the devel­op­ment of med­i­cines that target an unmet med­i­cal need. It is based on en­hanced inter­action be­tween sponsor com­pa­nies and the EMA to optimise devel­op­ment plans and speed up evaluation so these med­i­cines can reach patients earlier.[ii]

Orphan desig­na­tion may be granted for ther­a­pies in­tended to treat con­di­tions that affect fewer than 200,000 people in the U.S. The desig­na­tion qualifies the sponsor of the drug for var­i­ous devel­op­ment incentives of the Orphan Drug Act, in­­clud­ing tax credits for qualified clin­i­cal testing[iii].

In Europe, sponsors who obtain Orphan Desig­na­tion for a poten­tial new med­i­cine ben­e­fit from a range of incentives, in­­clud­ing protocol assistance, access to the centralised pro­ce­dure, mar­ket exclusivity and fee re­duc­tions.[iv]

About GSK2857916

GSK2857916 is a humanised anti BCMA mono­clonal anti­body con­ju­gated to the cyto­toxic agent mono­methyl auristatin-F, via non-cleavable linker (drug linker tech­nology in-licensed from Seattle Genetics).

GSK2857916 is cur­rently in phase 1 clin­i­cal devel­op­ment (NCT02064387) in patients with re­lapsed/​refractory mul­ti­ple myeloma and other ad­vanced haema­to­logic malig­nan­cies expressing BCMA.

GSK2857916 is not approved for use any­where in the world.

GSK - one of the world's lead­ing re­search-based pharma­ceu­tical and health­care com­pa­nies - is com­mit­ted to im­prov­ing the quality of human life by enabling people to do more, feel better and live longer. For fur­ther in­­for­ma­tion please visit www.gsk.com.

Cautionary state­ment re­gard­ing for­ward-looking state­ments

GSK cautions in­­vestors that any for­ward-looking state­ments or pro­jec­tions made by GSK, in­­clud­ing those made in this an­nouncement, are subject to risks and un­cer­tain­ties that may cause actual re­­sults to differ ma­teri­ally from those pro­jected. Such factors in­clude, but are not lim­ited to, those described under Item 3.D 'Principal risks and un­cer­tain­ties' in the com­pany's Annual Report on Form 20-F for 2016.

References

[i] U.S. Food and Drug Admin­istra­tion Frequently Asked Questions: Break­through ther­a­pies. Accessed Octo­ber 2017. Available at: https://www.fda.gov/regulatoryinformation/lawsenforcedbyfda/significantamendmentstothefdcact/fdasia/ucm341027.htm

[ii] Euro­pean Medicines Agency PRIME: priority med­i­cines. Accessed Octo­ber 2017. Available at: http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000660.jsp

[iii] U.S. Food and Drug Admin­istra­tion Designating an Orphan Product: Drugs and Biologic Products. Accessed Octo­ber 2017. Available at:https://www.fda.gov/forindustry/developingproductsforrarediseasesconditions/howtoapplyfororphanproductdesignation/default.htm

[iv] Euro­pean Medicines Agency Orphan Desig­na­tions. Accessed Octo­ber 2017. Available at: http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000029.jsp

Source: GlaxoSmithKline.