Novartis receives US Food and Drug Administration (FDA) Orphan Drug Designation for branaplam (LMI070) in Huntington’s disease (HD)

  • Huntington’s disease is a rare, inherited neurodegenerative disease that leads to progressive disability and death
  • There are no approved disease modifying therapies that delay disease onset or slow progression of the disease
  • Branaplam (LMI070) is an orally administered, small molecule RNA splicing modulator that could potentially reduce the levels of mutant huntingtin protein

Basel, October 21, 2020 — Novartis today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for branaplam (LMI070) in Huntington’s disease (HD). An Orphan Drug Designation grants special status to a drug that treats a rare disease or condition, and provides companies certain benefits to encourage the continued development of medicines that bring novel solutions to patients with these severe diseases.

In preclinical models, branaplam has been shown to reduce levels of mutant huntingtin protein. In addition, during the investigation of branaplam in spinal muscular atrophy (SMA), it was also observed to reduce huntingtin messenger RNA (mRNA) in SMA patients. A decrease of huntingtin mRNA is expected to result in reduction of huntingtin protein levels, the underlying cause of HD. Based on these findings, Novartis intends to start a development program for branaplam to determine if it has the potential to be a transformative treatment for people living with this devastating condition.

Current treatment options for HD are limited to symptomatic treatments and there are no approved disease modifying therapies that delay disease onset or slow progression of the disease.

Branaplam is currently under investigation for the treatment of spinal muscular atrophy (SMA). SMA is a rare, progressive genetic disease, characterized by loss of motor neurons that are responsible for muscle function. Branaplam is dosed once weekly for the treatment of SMA, and the same dosing regimen may also be a possibility for HD.

Novartis plans to start the Phase IIb trial for branaplam in HD patients in 2021.

About branaplam
Branaplam (LMI070) is a once-weekly, orally administered, small molecule RNA splicing modulator that is currently under investigation for the treatment of spinal muscular atrophy (SMA). SMA is a rare, progressive genetic disease, characterized by loss of motor neurons that are responsible for muscle function. Novartis has more than 5 years’ clinical experience with branaplam in our ongoing SMA development program. 

More:  Novartis announces MET inhibitor capmatinib (INC280), the first potential treatment for METex14 mutated advanced non-small cell lung cancer, granted priority FDA review

About Huntington’s disease
Huntington’s disease (HD) is a rare, inherited neurodegenerative disease that leads to progressive disability and death. Everyone has the huntingtin (HTT) gene, but only those who have a mutated form of the gene will develop the disease.

HD is characterized by progressive worsening in motor, cognitive and psychiatric symptoms. These symptoms usually appear between the ages of 30 to 50, and worsen over a 15-20-year period. Approximately 70,000 people across Europe and the US have been clinically diagnosed with HD.

Current treatment options for HD are limited to symptomatic treatments and there are no approved disease modifying therapies that delay disease onset or slow progression of the disease.

Disclaimer
This media update contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,” “plan,” “may,” “could,” “would,” “expect,” “anticipate,” “seek,” “look forward,” “believe,” “committed,” “investigational,” “pipeline,” “launch,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this media update, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations 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 the investigational or approved products described in this media update will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, 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 media update as of this date and does not undertake any obligation to update any forward-looking statements contained in this media update as a result of new information, future events or otherwise.

More:  Meet a visionary who beat the odds to realize his vision of inclusive eye care | Novartis

About Novartis
Novartis is reimagining medicine to improve and extend people’s lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 140 nationalities work at Novartis around the world. Find out more at
https://www.novartis.com.

Novartis is on Twitter. Sign up to follow @Novartis at https://twitter.com/novartisnews
For Novartis multimedia content, please visit https://www.novartis.com/news/media-library
For questions about the site or required registration, please contact [email protected]

# # #

Novartis Media Relations
E-mail: [email protected]

Antonio Ligi
Novartis External Communications
+41 61 324 1374 (direct)
[email protected]
Gonzalo Linares
Novartis Global Drug Development Communications & Patient Advocacy
T +41 61 324 5117
M +41 79 386 7432
[email protected]

Novartis Investor Relations
Central investor relations line: +41 61 324 7944
E-mail: [email protected]

Central North America 
Samir Shah+41 61 324 7944Sloan Simpson+1 862 778 5052
Thomas Hungerbuehler        
Isabella Zinck
+41 61 324 8425
+41 61 324 7188
  
    
    


More from: | Category: Pharmaceutical Company News