The NDA for LBH589 was submitted to the FDA in March 2014. In May 2014, the FDA granted priority review status to LBH589, reducing the standard 12-month review period to eight months. The extension to the LBH589 NDA review period follows an FDA Oncologic Drugs Advisory Committee (ODAC) meeting earlier this month.

"We are committed to work­ing with the FDA as they con­tinue to review the LBH589 NDA," said Alessandro Riva, MD, Global Head of Oncology Development and Medical Affairs, Novartis Oncology. "Multiple myeloma remains an incurable cancer where patients who have re­lapsed or become resistant to avail­able ther­a­pies need new treat­ment options."

About multiple myeloma and LBH589

Multiple myeloma is a cancer of the plasma cells, a kind of white blood cell present in bone marrow-the soft, blood-producing tissue that fills the center of most bones. The cancer is caused by the pro­duc­tion and growth of ab­nor­mal cells within the plasma, which multiply and build up in the bone marrow, pushing out healthy cells and preventing them from functioning normally[1]. Multiple myeloma is an incurable disease with a high rate of relapse (when the cancer returns) and patients often become refractory (unresponsive to ther­apy), despite cur­rently avail­able treat­ments[2]. It typically occurs in individuals 60 years of age or older, with few cases in individuals younger than 40[3].

Epigenetics is the cell pro­gramming that governs gene ex­pres­sion and cell devel­op­ment[4]. In multiple myeloma, the normal epigenetic process is disrupted (also called epigenetic dysregulation) resulting in the growth of can­cer­ous plasma cells, poten­tial resistance to current treat­ment and ultimately disease pro­gres­sion[5],[6].

LBH589 is a potent pan-deacetylase (pan-DAC) inhibitor that if approved could be a first-in-class treat­ment for patients with re­lapsed or re­lapsed and refractory multiple myeloma[7]. As an epigenetic regulator, LBH589 may help restore cell pro­gramming in multiple myeloma[8].

Because LBH589 is an inves­ti­ga­tional com­­pound, the safety and efficacy profile has not yet been estab­lish­ed. Access to this inves­ti­ga­tional com­­pound is avail­able only through carefully controlled and monitored clin­i­cal trials. These trials are designed to better under­stand the poten­tial benefits and risks of the com­­pound. Because of the uncertainty of clin­i­cal trials, there is no guar­an­tee that LBH589 will ever be commercially avail­able any­where in the world.

* Trade name Velcade^® registered to Millennium Pharma­ceu­ticals, Inc.

Disclaimer

The foregoing release con­tains forward-looking state­ments that can be identified by words such as "investigational," "committed," "continues," "could," "may," "yet," "will," or similar terms, or by express or implied discussions regarding poten­tial market­ing approvals for LBH589, or regarding poten­tial future revenues from LBH589. You should not place undue reliance on these state­ments. Such forward-looking state­ments are based on the current beliefs and ex­pec­ta­tions of man­agement regarding future events, and are subject to sig­nif­i­cant known and unknown risks and un­cer­tain­ties. Should one or more of these risks or un­cer­tain­ties materialize, or should under­lying assump­tions prove incorrect, actual results may vary materially from those set forth in the forward-looking state­ments. There can be no guar­an­tee that LBH589 will be approved for sale in any market where it has been submitted, or at any particular time. Neither can there be any guar­an­tee that LBH589 will be submitted or approved for sale in any addi­tional markets, or at any particular time. Nor can there be any guar­an­tee that LBH589 will be commercially successful in the future. In particular, man­agement's ex­pec­ta­tions regarding LBH589 could be affected by, among other things, the un­cer­tain­ties in­her­ent in research and devel­op­ment, in­­clud­ing unexpected clin­i­cal trial results and addi­tional analysis of existing clin­i­cal data; unexpected regu­la­tory actions or delays or gov­ern­ment reg­u­la­tion generally; the com­pany's ability to obtain or maintain pro­pri­e­tary intellectual property protection; general economic and industry con­di­tions; global trends to­ward health care cost con­tainment, in­­clud­ing ongoing pricing pressures; unexpected manu­fac­tur­ing issues, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Com­mis­sion. Novartis is providing the in­­for­ma­tion in this press release as of this date and does not under­take any obli­ga­tion to update any forward-looking state­ments con­tained in this press release as a result of new in­­for­ma­tion, future events or other­wise.

About Novartis 

Novartis provides inno­va­tive health­care solu­tions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis offers a di­vers­i­fied portfolio to best meet these needs: inno­va­tive medicines, eye care, cost-saving generic pharma­ceu­ticals, preventive vaccines, over-the-counter and animal health prod­ucts. Novartis is the only global com­pany with leading positions in these areas. In 2013, the Group achieved net sales of USD 57.9 billion, while R&D throughout the Group amounted to approx­i­mately USD 9.9 billion (USD 9.6 billion excluding im­pair­ment and amortization charges). Novartis Group com­pa­nies employ approx­i­mately 133,000 full-time-equivalent asso­ci­ates and sell prod­ucts in more than 150 countries around the world. For more in­­for­ma­tion, please visit www.novartis.com.

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References
[1] American Cancer Society. Multiple Myeloma. Available at: www.cancer.org/acs/groups/cid/documents/webcontent/003121-pdf.pdf. Accessed November 2014
[2] The Leukemia and Lymphoma Society. Myeloma. Revised 2013;1:48.
[3] National Cancer Institute. SEER Stat Fact Sheets: Myeloma. Available at: seer.cancer.gov/statfacts/html/mulmy.html. Accessed November 2014.
[4] Grønbæk K, Treppendahl M, Asmarand F, Guldberg P. Epigenetic Changes in Cancer as Potential Targets for Prophylaxis and Maintenance Therapy. Basic & Clinical Pharmacology & Toxicology. 2008;103:389-396.
[5] Smith EM, Boyd K, Davies FE. The Potential Role of Epigenetic Therapy in Multiple Myeloma. Br J Haematol. 2009;148:702-713.
[6] Muntean AG, Hess JL. Epigenetic Dysregulation in Cancer. Am J Pathol.2009;175:1353-1361.
[7] San-Miguel J, et al. Randomized Phase III Trial of Panobinostat Plus Bortezomib and Dexamethasone Versus Placebo Plus Bortezomib and Dexamethasone in Relapsed or Relapsed and Refractory Multiple Myeloma. The Lancet Oncology. 2014.
[8] Maes K, et al. Epigenetic Modulating Agents as a New Therapeutic Approach in Multiple Myeloma. Cancers. 2013;5:430-461.

Source: Novartis.