Orphan Drug Act Fueling $200 Billion Dollar Market

PALM BEACH, Florida, April 23, 2020 /PRNewswire/ -- The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the treatment, prevention or diagnosis of a rare disease or condition, which is one that affects less than 200,000 persons in the US or meets cost recovery provisions of the act. The loophole was that since these are small percentages of prospective patients, it is hard for developers to recoup the cost of research, so the companies were granted a 7 year exclusivity, but the Congress reaffirmed and recognized the need to continue supporting research and development for rare diseases. A report from Evaluate.com stated: "Big pharma's recent dominance of the orphan market has fueled calls to reform the orphan drug act in the US. Regardless, orphan drug sales are forecast to increase from $119 Billion in 2018 to $217 Billion in 2024. Watch this year's report into the top performing drugs, companies and sales of the orphan market. Active biotech and pharma companies in the markets this week include CNS Pharmaceuticals, Inc. (NASDAQ: CNSP), Verastem, Inc. (NASDAQ: VSTM), Mallinckrodt plc (NYSE: MNK), AbbVie Inc. (NYSE: ABBV), Bristol-Myers Squibb Company (NYSE: BMY).

Four decades ago, patients with rare diseases had very few treatment options. The 1983 Orphan Drug Act changed everything by providing substantial tax incentives for pharmaceutical companies to invest in rare conditions affecting fewer than 200,000 Americans. With an estimated 7,000 orphan diseases, one out of every 10 Americans lives with a rare condition. After nearly four decades of relatively consistent growth, orphan drugs now comprise a cornerstone of the pharmaceutical market, with this category predicted to rake in more than 18% of overall prescription sales by 2024. Remarkably, the market is so robust that rare diseases now take home a large portion of FDA drug approvals. In 2019, just under half (44%) of new FDA approvals went to orphan drugs.

CNS Pharmaceuticals, Inc. (NASDAQ: CNSP) BREAKING NEWS: CNS Pharmaceuticals Announces Filing of FDA Orphan Drug Designation for Brain Cancer Drug Berubicin - CNS Pharmaceuticals, a biotechnology company specializing in the development of novel treatments for brain tumors, today announced it has filed an application with the U.S. Food and Drug Administration (FDA) to receive Orphan Drug Designation (ODD) for its lead product Berubicin.

Under a prior developer, Berubicin, then known as RTA 744, was granted ODD by the FDA for the treatment of malignant gliomas. In the prior developer's Phase 1 trial of Berubicin, 44% of the patients demonstrated a significant improvement in progression free survival. Additionally, one patient in this study experienced a complete response to his treatment with Berubicin.

"We are excited to announce the Orphan Drug application submission for Berubicin, as it would grant special status and accelerate the development of Berubicin to treat glioblastoma, one of the world's most aggressive forms of cancer," stated John Climaco, CEO of CNS Pharmaceuticals. "We are pleased to continue to execute upon our strategic initiatives and submit our application within the anticipated timeline outlined within our previous filings. We feel cautiously optimistic about the application, given the past Orphan Drug Designation of the molecule and positive Phase 1 results. We look forward to initiating a Phase II trial evaluating the effect of Berubicin on patients with glioblastoma later this year."

The Orphan Drug Act ("ODA") provides for granting special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor. This status is referred to as orphan designation (or sometimes "orphan status").

The FDA grants Orphan Drug Designation status to products that treat rare diseases, providing incentives to sponsors developing drugs or biologics. The FDA defines rare diseases as those affecting fewer than 200,000 people in the United States at any given time. Due to small patient numbers, treatment for these rare diseases would not be considered economically feasible without government programs to support their economic viability. Orphan Drug Designation would qualify Berubicin for certain benefits and incentives, including seven years of marketing exclusivity if regulatory approval is ultimately received for the designated indication, potential tax credits for certain activities, eligibility for orphan drug grants, and the waiver of certain administrative fees. The receipt of Orphan Drug Designation status does not change the regulatory requirements or process for obtaining marketing approval. Read this and more news for CNSP at:  https://www.financialnewsmedia.com/news-cnsp/

Other recent developments in the biotech industry include:

Verastem, Inc. (NASDAQ: VSTM) recently  announced that an abstract highlighting preliminary results from the ongoing investigator-initiated clinical study investigating VS-6766, its RAF/MEK inhibitor, in combination with defactinib, its FAK inhibitor, in patients with KRAS mutant advanced solid tumors has been selected for a virtual poster presentation at the upcoming American Association for Cancer Research (AACR) 2020 Virtual Annual Meeting I, taking place April 27-28, 2020.

This ongoing study is an open label, dose escalation and expansion study. The expansion cohorts are currently ongoing in patients with KRAS mutant advanced solid tumors, including low grade serous ovarian cancer (LGSOC), non-small cell lung cancer (NSCLC) and colorectal cancer (CRC). Following the virtual data presentation, Verastem Oncology will host an investor conference call to discuss the presented data. The exact date and time of the investor conference call will be announced soon.

"These early clinical results led to our decision to in-license VS-6766 earlier this year and accelerate development of this exciting combination program for patients with KRAS mutant cancers," said Brian Stuglik, Chief Executive Officer of Verastem Oncology. "The synergy between VS-6766 and defactinib has been encouraging and we look forward to sharing the data with the medical and scientific communities later this month."

Mallinckrodt plc (NYSE: MNK) recently announced that the U.S. Food and Drug Administration (FDA) has accepted for review the company's New Drug Application (NDA) for terlipressin, an investigational agent being evaluated for the treatment of hepatorenal syndrome type 1 (HRS-1). On March 17, 2020 the company announced the completion of its rolling submission of the NDA for terlipressin. The FDA assigned a Prescription Drug User Fee Act (PDUFA) target date of September 12, 2020.

HRS-1 is an acute and life-threatening syndrome involving acute kidney failure in people with cirrhosis. The condition has a median survival time of approximately two weeks and greater than 80 percent mortality within three months if left untreated. At present, there are no approved drug therapies for HRS-1 in the U.S., and it is estimated to affect between 30,000 and 40,000 patients in the U.S. annually.

AbbVie Inc. (NYSE: ABBV) recently announced that the U.S. Food and Drug Administration (FDA) approved the use of IMBRUVICA^® (ibrutinib) in combination with rituximab for the treatment of previously untreated patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). This milestone marks the 11th FDA approval for IMBRUVICA since it was first approved in 2013 and the sixth in CLL, the most common form of leukemia in adults. 

"The gold-standard first-line treatment option for many patients with chronic lymphocytic leukemia who were fit enough to tolerate an aggressive treatment course had been the intravenous chemoimmunotherapy of FCR – that is, until today," said Brian Koffman, M.D., C.M., Chief Medical Officer and Executive Vice President, CLL Society. "The FDA approval of ibrutinib and rituximab regimen is welcome news for these previously untreated patients who have been looking forward to a non-chemotherapy treatment option. The results from ECOG-ACRIN's E1912 clinical trial in previously untreated, younger adult patients and today's milestone represent a paradigm shift in how physicians can treat patients with CLL and may enable many to choose a non-chemotherapy treatment option."

Bristol-Myers Squibb Company (NYSE: BMY) recently announced that CheckMate -743, a pivotal Phase 3 trial evaluating Opdivo^® (nivolumab) in combination with Yervoy^® (ipilimumab) in previously untreated malignant pleural mesothelioma (MPM) met its primary endpoint of overall survival (OS). Based on a pre-specified interim analysis conducted by the independent Data Monitoring Committee, Opdivo in combination with Yervoy resulted in a statistically significant and clinically meaningful improvement in OS compared to chemotherapy (pemetrexed and cisplatin or carboplatin).

The safety profile of Opdivo plus Yervoy observed in the trial reflects the known safety profile of the combination. "Malignant pleural mesothelioma is a devastating disease that has seen limited treatment advances over the past decade," said Sabine Maier, M.D., development lead, thoracic cancers, Bristol Myers Squibb. "These topline results from the CheckMate -743 trial demonstrate the potential of Opdivo plus Yervoy in previously untreated patients with malignant pleural mesothelioma, and is another example of the established efficacy and safety of the dual immunotherapy combination seen in multiple tumor types. We would like to thank the patients who participated in this trial, as well as the investigators and site personnel for their perseverance during the conduct of this study and in delivering this important result for patients in the midst of the COVID-19 pandemic. We look forward to working with investigators to present the results at a future medical meeting, and to discussing them with health authorities."

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