If approved, dara­tu­mu­mab will offer a new option for double refractory, heavily pre-treated patients

Raritan, NJ (Press Release) – Janssen Research & Development, LLC (Janssen) announced today the U.S. Food and Drug Admin­istra­tion (FDA) has accepted for Priority Review the Biologics License Application (BLA) for dara­tu­mu­mab as a treat­ment for patients with multiple myeloma who are refractory to both a pro­tea­some inhibitor (PI) and an immuno­modu­la­tory agent (IMiD), or who have received three or more prior lines of ther­apy, in­­clud­ing a PI and an IMiD. This is referred to as "double refractory" multiple myeloma, which occurs when a patient's disease has be­come resistant to at least two of the most commonly utilized and active classes of anti-myeloma agents. Dara­tu­mu­mab, an inves­ti­ga­tional human anti-CD38 mono­clonal anti­body, received Break­through Therapy Desig­na­tion from the FDA for this patient pop­u­la­tion in May 2013.

The FDA grants Priority Review to inves­ti­ga­tional ther­a­pies that if approved, may offer sig­nif­i­cant im­prove­ments in the treat­ment, prevention or diag­nosis of a serious con­di­tion.¹ This desig­na­tion shortens the review period to six months com­pared to 10 months for Standard Review. With today's announcement, the FDA has assigned a Prescription Drug User Fee Act (PDUFA) target date of March 9, 2016 to render a de­ci­sion on the dara­tu­mu­mab appli­ca­tion.

"Daratumumab has the poten­tial to be the first anti-CD38 mono­clonal anti­body approved to treat multiple myeloma, offering these patients an im­por­tant new treat­ment path­way," said Craig Tendler, M.D., Vice Pres­i­dent, Late Development and Global Medical Affairs, Oncology, Janssen. "The FDA's acceptance of our appli­ca­tion for dara­tu­mu­mab marks an im­por­tant step for people affected by multiple myeloma. We look for­ward to work­ing with the FDA during this Priority Review."

The regu­la­tory sub­mission for dara­tu­mu­mab, which was ini­ti­ated on June 5, 2015, is primarily sup­ported by data from the Phase 2 MMY2002 (SIRIUS) mono­therapy study, which were presented in May/June 2015 at the 51^st Annual Meeting of the American Society of Clinical Oncology (ASCO). Additional data from four other studies, in­­clud­ing the Phase 1/2 GEN501 mono­therapy study, recently published in The New England Journal of Medicine, also sup­port the sub­mission. Dara­tu­mu­mab is the second med­i­cine in the Janssen on­col­ogy portfolio to receive Break­through Therapy Desig­na­tion, which is in­tended to expedite the de­vel­op­ment and review time for a poten­tial new med­i­cine.

In August 2012, Janssen Biotech, Inc. and Genmab A/S entered a world­wide agree­ment that granted Janssen an exclusive license to develop, manu­fac­ture and com­mer­cialize dara­tu­mu­mab. Janssen is the global sponsor of all but one clin­i­cal studies for dara­tu­mu­mab. If approved, dara­tu­mu­mab would be com­mer­cialized in the U.S. by Janssen Biotech, Inc.

About Multiple Myeloma

Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is char­ac­ter­ized by an excess proliferation of plasma cells.² Multiple myeloma is the third most common blood cancer in the U.S., behind only leukemia and lym­phoma.³ Approximately 26,850 new patients will be diag­nosed with multiple myeloma, and approx­i­mately 11,240 people will die from the disease in the U.S. in 2015.⁴ Globally, it is esti­mated that 124,225 people will be diag­nosed and 87,084 will die from the disease in 2015.5,⁶ While some patients with multiple myeloma have no symp­toms at all, most patients are diag­nosed due to symp­toms which can in­clude bone problems, low blood counts, cal­cium elevation, kidney problems or in­fec­tions.⁷ Patients who relapse after treat­ment with standard ther­a­pies, in­­clud­ing PIs or IMiDs, have poor prognoses and few treat­ment options.⁸

About Dara­tu­mu­mab

Daratumumab is an inves­ti­ga­tional human mono­clonal anti­body (mAb) that binds with high affinity to the CD38 molecule, which is highly ex­pressed on the surface of multiple myeloma cells. It is believed to induce rapid tumor cell death through multiple immune-mediated mech­a­nisms,⁹ in­­clud­ing complement-dependent cyto­tox­icity,¹⁰ anti­body-dependent cellular phago­cytosis¹¹ and anti­body-dependent cellular cyto­tox­icity,¹² as well as via induction of apop­tosis.¹³ Five Phase 3 clin­i­cal studies with dara­tu­mu­mab in re­lapsed and front­line settings are cur­rently ongoing. Additional studies are ongoing or planned to assess its poten­tial in other malignant and pre-malignant diseases on which CD38 is ex­pressed, such as smol­der­ing myeloma and non-Hodgkin lym­phoma.

About Janssen Research & Development, LLC

At Janssen, we are dedicated to addressing and solving some of the most im­por­tant unmet medical needs of our time in on­col­ogy, immunology, neuroscience, infectious diseases and vaccines, and cardiovascular and metabolic diseases. Driven by our commitment to patients, we develop inno­va­tive prod­ucts, services and health­care solu­tions to help people through­out the world. Janssen Research & Development, LLC and Janssen Biotech, Inc. are part of the Janssen Pharma­ceu­tical Com­panies of Johnson & Johnson. Please visit www.janssenrnd.com for more in­­for­ma­tion.

Janssen in Oncology

In on­col­ogy, our goal is to fundamentally alter the way cancer is under­stood, diag­nosed and man­aged, reinforcing our commitment to the patients who in­spire us. In looking to find inno­va­tive ways to address the cancer chal­lenge, our pri­mary efforts focus on several treat­ment and prevention solu­tions. These in­clude a focus on hema­to­logic malig­nan­cies, prostate cancer and lung cancer; cancer interception with the goal of devel­op­ing prod­ucts that interrupt the carcinogenic process; bio­­markers that may help guide targeted, individualized use of our ther­a­pies; as well as safe and effective identi­fi­ca­tion and treat­ment of early changes in the tumor microenvironment. Please visit on­col­ogy.janssenrnd.com.

This press release con­tains "forward-looking state­ments" as defined in the Private Se­cu­ri­ties Lit­i­ga­tion Reform Act of 1995 re­gard­ing prod­uct devel­op­ment. The reader is cautioned not to rely on these for­ward-looking state­ments. These state­ments are based on current ex­pec­ta­tions of future events. If under­lying assump­tions prove inaccurate or known or unknown risks or un­cer­tain­ties ma­teri­alize, actual results could vary ma­teri­ally from the ex­pec­ta­tions and projections of Janssen Research & Development, LLC and/or Johnson & Johnson. Risks and un­cer­tain­ties in­clude, but are not limited to: chal­lenges and un­cer­tain­ties in­her­ent in new prod­uct devel­op­ment, in­­clud­ing the uncertainty of clin­i­cal success and of obtaining regu­la­tory approvals; com­pe­ti­tion, in­­clud­ing technological ad­vances, new prod­ucts and patents attained by com­pet­i­tors; chal­lenges to patents; changes to appli­­cable laws and reg­u­la­tions, in­­clud­ing global health care reforms; and trends to­ward health care cost con­tainment. A further list and description of these risks, un­cer­tain­ties and other factors can be found in Johnson & Johnson's Annual Report on Form 10-K for the fiscal year ended December 28, 2014, in­­clud­ing in Exhibit 99 thereto, and the com­pany's sub­se­quent filings with the Se­cu­ri­ties and Exchange Com­mis­sion. Copies of these filings are avail­able online at www.sec.gov, www.jnj.com or on request from Johnson & Johnson. None of the Janssen Pharma­ceu­tical Com­panies or Johnson & Johnson under­takes to update any for­ward-looking state­ment as a result of new in­­for­ma­tion or future events or devel­op­ments.

References

¹ U.S. Food and Drug Admin­istra­tion. "Priority Review." Available at http://www.fda.gov/forpatients/approvals/fast/ucm405405.htm. Accessed September 2015.
² American Cancer Society. "Multiple Myeloma Overview." Available at http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-what-is-multiple-myeloma. Accessed September 2015.
³ National Cancer Institute. "A Snapshot of Myeloma." Available at www.cancer.gov/research/progress/snapshots/myeloma. Accessed September 2015.
⁴ American Cancer Society. "What are the key statistics about multiple myeloma?" http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-key-statistics. Accessed September 2015.
⁵ GLOBOCAN 2012: Estimated Cancer Incidence, Mortality and Prevalence Worldwide: Number of New Cancers in 2015. Available at: http://globocan.iarc.fr/old/burden.asp?selection_pop=224900&Text-p=World&selection_cancer=17270&Text-c=Multiple+myeloma&pYear=3&type=0&window=1&submit=%C2%A0Execute. Accessed August 2015.
⁶ GLOBOCAN 2012: Estimated Cancer Incidence, Mortality and Prevalence Worldwide: Number of Cancer Deaths in 2015. Available at http://globocan.iarc.fr/old/burden.asp?selection_pop=224900&Text-p=World&selection_cancer=17270&Text-c=Multiple+myeloma&pYear=3&type=1&window=1&submit=%C2%A0Execute. Accessed August 2015.
⁷ American Cancer Society. "How is Multiple Myeloma Diagnosed?" http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-diagnosis. Accessed September 2015.
⁸ Kumar, SK et al. Leukemia. 2012 Jan;26(1):149-57.
⁹ Michael de Weers et al. Dara­tu­mu­mab, a Novel Therapeutic Human CD38 Monoclonal Antibody, Induces Killing of Multiple Myeloma and Other Hematological Tumors. The Journal of Immunology. February 1, 2011. Vol. 186, No. 3 1840-1848.
¹⁰ Michael de Weers et al. Dara­tu­mu­mab, a Novel Therapeutic Human CD38 Monoclonal Antibody, Induces Killing of Multiple Myeloma and Other Hematological Tumors. The Journal of Immunology. February 1, 2011. Vol. 186, No. 3 1840-1848.
¹¹ Yulian Khagi and Tomer M Mark. Potential role of dara­tu­mu­mab in the treat­ment of multiple myeloma. Onco Targets Ther. 2014; 7: 1095–1100.
¹² Michael de Weers et al. Dara­tu­mu­mab, a Novel Therapeutic Human CD38 Monoclonal Antibody, Induces Killing of Multiple Myeloma and Other Hematological Tumors. The Journal of Immunology. February 1, 2011. Vol. 186 no. 3 1840-1848.
¹³ Jansen JH, et al. Blood. 2012; 120.2974.

Source: Janssen Research & Development, LLC.