CTI: FDA Review Period Extended for Pacritinib in Myelofibrosis

 CTI BioPharma Corp. (Nasdaq: CTIC) today announced the U.S. Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) for pacritinib for the treatment of adult patients with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis (MF) with a baseline platelet count of <50 × 109/L. The Prescription Drug User Fee Act (PDUFA) action date has been extended by three months to February 28, 2022.

In the second quarter of 2021, the FDA granted priority review for CTI's NDA for patients with myelofibrosis with a PDUFA date of November 30, 2021. In the course of product labeling discussions, the FDA requested additional clinical data, which was submitted to the agency on November 24, 2021. Earlier today, the FDA informed the Company that it considers the data submission to constitute a "major amendment" to the NDA and therefore the PDUFA date has been extended by three months to provide additional time for a full review of the submission. At the current time, CTI is not aware of any major deficiencies in the application.

"CTI is continuing to engage collaboratively and constructively with the FDA during review of our NDA," said Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI Biopharma. "We are committed to providing patients suffering from cytopenic myelofibrosis with a new treatment option as soon as possible and are confident in pacritinib's potential to establish a new standard of care."

Pacritinib is a novel oral kinase inhibitor with specificity for JAK2, IRAK1 and CSF1R, without inhibiting JAK1. The NDA was accepted based on the data from the Phase 3 PERSIST-2 and PERSIST-1 and the Phase 2 PAC203 clinical trials, with a focus on the severely thrombocytopenic (platelet counts less than 50 x 109/L) patients enrolled in these studies who received pacritinib 200 mg twice a day, including both frontline treatment-naive patients and patients with prior exposure to JAK2 inhibitors. In the PERSIST-2 study, in patients with severe thrombocytopenia who were treated with pacritinib 200 mg twice a day, 29% of patients had a reduction in spleen volume of at least 35%, compared to 3% of patients receiving the best available therapy, which included ruxolitinib; 23% of patients had a reduction in total symptom scores of at least 50%, compared to 13% of patients receiving the best available therapy. In the same population of patients treated with pacritinib, adverse events were generally low grade, manageable with supportive care, and rarely led to discontinuation. Platelet counts and hemoglobin levels were also stabilized.

https://www.prnewswire.com/news-releases/cti-biopharma-announces-extension-of-fda-review-period-for-pacritinib-in-myelofibrosis-with-severe-thrombocytopenia-301434498.html

FDA eyes rapid review for omicron vaccines, drugs

 The Food and Drug Administration (FDA) is reportedly eyeing steps for an expedited review of vaccines and drugs targeting the newly detected omicron variant should such steps be necessary.

The Wall Street Journal, citing people familiar with the matter, reported Friday that data and study guidelines are being established to expedite reviews of drugs and vaccines. The people also told the newspaper that the agency was meeting with drugmakers, too.

The FDA is reportedly setting up parameters around what studies could look like for omicron vaccine and drug approval. In the case of vaccines, drugmakers could be allowed to look at hundreds of subjects and their immune responses compared to vaccine test trials that wait for COVID-19 to be detected and include thousands of participants, according to the Journal.

One person familiar with the matter told the newspaper that the development and testing of new vaccines would only need three months. Following that, the FDA would take between a week or two to make a decision on authorization of the vaccine following a rapid review process.

"We could probably get a good bit of mileage just from boosting with the ancestral strain vaccine that we already have," White House chief medical adviser Anthony Fauci said Friday. 

But he added the administration is also working with pharmaceutical companies to develop multiple contingency plans, including increasing production of current vaccines, developing a vaccine that's effective against both the ancestral strain and a new variant, or developing a variant-specific booster.

The FDA pointed to existing guidance for manufacturers, which was last updated in the spring, to understand how the agency will evaluate possible new vaccines. FDA also noted a statement from acting Commissioner Janet Woodcock issued on Monday about the omicron variant.

"Historically, the work to obtain the genetic information and patient samples for variants and then perform the testing needed to evaluate their impact takes time. However, we expect the vast majority of this work to be completed in the coming weeks," she said.

The new reported steps come as health authorities race to learn more about the omicron variant, which has been detected in more than 30 countries including the U.S. after being discovered by scientists in South Africa.

On Monday, the Biden administration began enforcing travel restrictions on eight southern African nations in an effort to curb the spread of the variant.

Officials from the World Health Organization said on Friday that they believed that the same steps taken during the delta wave would be sufficient in tackling the omicron variant, including a renewed push on vaccinating people, wearing facial coverings and social distancing. 

“The positive news in all of this is that none of the information we have currently about omicron suggests we need to change the directions of our response,” WHO regional director for the Western Pacific, Takeshi Kasai, said during a news conference.

https://thehill.com/policy/healthcare/584214-fda-eyes-rapid-review-for-omicron-vaccines-drugs-report

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S.Africa COVID-19 cases triple in three days amid omicron

 New COVID-19 cases in South Africa have tripled in three days, according to new figures released by the country's health ministry on Thursday, sparking concerns over the spread of the new omicron variant.

South Africa is entering its fourth wave of COVID-19 infections due to the omicron variant, Phaahla added at a media briefing on Friday.

He said that the infections with the new variant have been detected in seven out of the country's nine provinces. Since omicron was first detected in the country eight days ago, cases have risen steadily, he said on Friday.

In the last 24 hours, there were 11,535 new cases recorded in South Africa, according to the National Institute for Communicable Diseases.

“Today, as we enter the fourth wave with a new variant, we can see confirmation of this warning. We have moved from a total of around 2,465 new cases last Thursday when this variant was announced to yesterday’s high of 11,535. A rise of just over 9,000 cases per day within seven days," he said.

The data showed a 22.4 percent positivity rate of people tested for the virus, up from 16.5 percent on Wednesday, a massive jump from a 1 percent positivity rate in early November, The Washington Post reported. 

"We can still manage this in a manner where government doesn't have to invoke serious restrictions over the next few days if we all just do our basic duties of the safety measures, but also if more and more of us who are eligible... approach their nearest vaccination sites," Phaahla said.

The omicron variant was announced by scientists last week in South Africa and has since been discovered in more than 20 countries.

The U.S. has already restricted travel from South Africa, Botswana, Zimbabwe, Namibia, Lesotho, Eswatini, Mozambique and Malawi in response to the new variant.

New omicron variant cases in the U.S. have been detected in five states — California, Minnesota, Colorado, New York and Hawaii.

https://thehill.com/policy/international/africa/584177-south-africa-covid-19-cases-triple-in-three-days-amid-omicron

Longeveron Shares Gain After Lead Product Gets FDA Orphan Drug Tag

 The FDA has granted Orphan Drug designation to Longeveron Inc's 

LGVN +0%

 (Get Free Alerts for LGVN) lead investigational product Lomecel-B for Hypoplastic left heart syndrome (HLHS).

• Lomecel-B is a cell-based therapy product derived from culture-expanded medicinal signaling cells (MSCs), sourced from the bone marrow of young, healthy adult donors. 
• See here Benzinga's Full FDA Calendar.
• HLHS is a congenital disability that affects normal blood flow through the heart. As the baby develops during pregnancy, the left side of the heart does not form correctly. 
• Hypoplastic left heart syndrome is one type of congenital heart defect.
• Last month, The FDA granted Rare Pediatric Disease (RPD) designation Lomecel-B for Hypoplastic Left Heart Syndrome.

https://www.benzinga.com/general/biotech/21/12/24426342/longeveron-shares-gain-after-lead-product-gets-fda-orphan-drug-tag-for-type-of-congenital-heart-d

Innovent, Lilly: Sintilimab in China National Reimbursement Drug List for 3 Indications

 Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high quality medicines for the treatment of oncology, metabolic, autoimmune and other major diseases, and Eli Lilly and Company ("Lilly") (NYSE: LLY), announce that the innovative PD-1 inhibitor sintilimab has been successfully included in the updated National Reimbursement Drug List ("NRDL") for all approved indications, according to the latest announcement from the China National Healthcare Security Administration ("NHSA"). The updated NRDL will officially take effect on January 1, 2022.

A total of four approved indications for sintilimab are now included in the updated NRDL:

• Three indications for sintilimab have been included in the NRDL for the first time, as follows: in combination with pemetrexed and platinum chemotherapy for the first-line treatment of advanced or recurrent nonsquamous non-small cell lung cancer (nsq NSCLC) without sensitizing EGFR mutations or ALK rearrangements; in combination with gemcitabine and platinum chemotherapy for the first-line treatment of advanced or recurrent squamous non-small cell lung cancer (sq NSCLC); and in combination with BYVASDA® (bevacizumab biosimilar injection) for the first-line treatment of unresectable or advanced hepatocellular carcinoma (HCC).
• An indication for relapsed or refractory classic Hodgkin's lymphoma (cHL) after two lines or later of systemic chemotherapy, which was first included in the NRDL in 2019, has been successfully renewed this year.

https://www.biospace.com/article/releases/innovent-and-lilly-announce-successful-expansion-of-sintilimab-in-china-national-reimbursement-drug-list-to-include-three-additional-first-line-indications/