EMA accepts Aerie Pharma’s Roclanda application

The European Medicines Agency (EMA) accepts for review Aerie Pharmaceuticals’ (NASDAQ:AERI) marketing application for Roclanda (netarsudil and latanoprost ophthalmic solution) 0.02%/0.005% for the reduction of elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension.

The advisory group CHMP should issue an opinion in Q4.

The Phase 3 Mercury 3 study comparing Roclanda to the leading fixed-dose combo product in Europe should be completed in H2, the results of which will support pricing and reimbursement.

https://seekingalpha.com/news/3529057-ema-accepts-aerie-pharmas-roclanda-application

European Medicines Agency Accepts Innate Pharma Leukemia Submission

Innate Pharma SA (Euronext Paris: IPH – ISIN: FR0010331421; Nasdaq: IPHA) (“Innate” or the “Company”) today announced that the European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) for Lumoxiti^® (moxetumomab pasudotox-tdfk), a first-in-class medicine indicated for adult patients with relapsed or refractory hairy cell leukemia (HCL) who have received at least two prior systemic therapies, including treatment with a purine nucleoside analog.

“If approved by the EMA, Lumoxiti will be the first treatment available in Europe for relapsed or refractory hairy cell leukemia patients in more than twenty years, potentially changing the standard of care for these patients,” commented Pierre Dodion, MD, Executive Vice President and Chief Medical Officer of Innate Pharma. “We are dedicated in addressing the unmet need in this rare form of cancer that can result in serious and life-threatening conditions, and as such, are hopeful we can bring this important medicine to patients in Europe as soon as possible.”

The EMA filing is based on the final analysis of the pivotal Phase III trial of Lumoxiti, presented at ASH 2019^*. These data showed that 36 percent (29/80) of the relapsed or refractory hairy cell leukemia patients achieved durable complete response, defined as a CR with a hematological remission maintained for at least 180 days. The objective response rate (ORR) was at 75 percent. Eighty-one percent of patients with CR experienced eradication of minimal residual disease as reflected by MRD-negative status. In addition, there was a 61 percent probability that patients who achieved a CR would maintain it after five years.

The EMA filling acceptance follows the U.S. Food and Drug Administration (FDA) approval of Lumoxiti in September 2018.

https://www.biospace.com/article/releases/the-european-medicines-agency-accepts-the-regulatory-submission-for-lumoxiti-in-relapsed-or-refractory-hairy-cell-leukemia/

Mesoblast Starts Efficacy, Safety Data Submission to FDA for Graft-Host Med

Mesoblast Limited MESO 0.41% announced that the United States Food and Drug Administration (US FDA) has confirmed receipt of Mesoblast’s filing of clinical efficacy and safety data for remestemcel-L in its rolling Biologics License Application (BLA) for the treatment of children with steroid-refractory acute graft versus host disease (SR-aGVHD). The final module will be filed during January, and Mesoblast will request an expedited FDA review of the BLA under the product candidate’s existing Fast Track designation. If approved, remestemcel-L is planned to be launched in the US in 2020.

The clinical submission included analyses of 309 children with SR-aGVHD who have received remestemcel-L across three separate studies. In addition, Mesoblast provided new data in control pediatric subjects from the contemporaneous database of the Mount Sinai Acute GVHD International Consortium (MAGIC) to provide an unbiased and independent estimate of response rates and outcomes in matched pediatric control patients treated with institutional standard of care.

The results of the comparative analysis between Mesoblast’s open-label Phase 3 study and contemporaneous controls receiving institutional standard of care demonstrate the effectiveness of remestemcel-L in this patient population, with particular efficacy and survival benefit in patients with the most severe forms of aGVHD. These conclusions are supported by prior results from an Expanded Access Program in 241 children where remestemcel-L was used as salvage therapy after failure of steroids and other agents.

Acute GVHD is a potentially life-threatening condition which occurs in about 50% of patients who receive an allogeneic bone marrow transplant. Over 30,000 patients worldwide receive an allogeneic bone marrow transplant, primarily during treatment for blood cancers, and 20-25% are children1. Mortality at 12 months is as high as 90% in those with aGVHD and Grade C/D or III/IV disease severity2,3, and there are no approved treatments in the US for children under 12.

https://www.benzinga.com/news/20/01/15059323/mesoblast-submits-clinical-efficacy-and-safety-data-to-fda-in-rolling-biologics-license-application

Biotech Investors: Mark Your Calendar For These January PDUFA Dates

In a final push before the closing of the year, the FDA deemed it fit to clear even long pending ones, including Flexion Therapeutics Inc’s FLXN sNDA for the updation of the label for its osteoarthritis drug Zilretaa.

About seven new molecular entities, or NMEs, were approved in the month, including Allergan plc’s AGN migraine treatment Ubrelvy and Sarepta Therapeutics Inc’s SRPT Duchenne muscular dystrophy drug Vyondys 53.

A NME is a drug that contains an active ingredient, which hasn’t been approved by the FDA previously, either as a single ingredient drug or as part of a combination product. The number of NME approvals is considered a measure of innovation among biotechs.

NME approvals for the year totaled 48, a sizable number, although paling in significance before 2018’s 59.

There was rejection too, with the FDA turning down Correvio Pharma Corp’s CORV atrial fibrillation drug Brinavess.

PDUFA dates are deadlines for the FDA to review new drugs. The FDA is normally given 10 months to review new drugs. If a drug is selected for priority review, the FDA is allotted six months to review the drug. These time frames begin on the date that an NDA is accepted by the FDA as complete.

Here’re the key PDUFA dates for the first month of the new year.

Epizyme Awaits FDA Verdict On Cancer Drug After Positive Adcom Vote

• Company: Epizyme Inc EPZM
• Type of Application: NDA
• Candidate: tazemetostat
• Indication: metastatic or locally advanced, unresectable epithelioid sarcoma
• Date: Jan. 23

The NDA was accepted for accelerated review July 25, and ahead of the decision, the application was reviewed by FDA’s Oncologic Drugs Advisory Committee Dec. 18. Despite expectations for a negative verdict, the panel unanimously recommended approval of the drug.

Go Or No Go For Merck’s Antibacterial Drug

• Company: Merck & Co., Inc. MRK
• Type of Application: NDA & sNDA
• Candidate: DIFICID (fidaxomicin)
• Indication: Clostridium difficile infections
• Date: Jan. 24

Merck said Oct. 2 the FDA accepted an NDA for oral suspension formulation and a sNDA for tablets and oral suspension for the treatment of Clostridium difficile infections in children aged six months or older.

Difficid is a macrolide antibiotic medicine indicated to treat Clostridium difficile-associated diarrhea in adults, ages 18 years or older.

Can Aimmune Peanut Allergy Drug Win FDA Nod?

• Company: Aimmune Therapeutics Inc AIMT
• Type of Application: NDA
• Candidate: Palforzia (AR101)
• Indication: peanut allergy
• Date: late January

FDA’s Allergenic Products Advisory Committee voted 7-to-2 in favor of the efficacy of Palforzia and 8-to-1 in favor of its safety. Palforzia is a biologic oral immunotherapy being evaluated to treat peanut allergy in patients, aged 4 through 17 years.

In an interview with Benzinga following the Adcom verdict, CFO Eric Bjerkholt said the company is gearing for a commercial launch in about one to two weeks following the approval, and gave a tentative timeframe of late January or early February.

Adcom Meetings

A joint meeting of the FDA’s Anesthetic and Analgesic Drug Products Advisory Committee and the Drug Safety and Risk Management Advisory Committee will discuss Jan. 14, Nektar Therapeutics’s NKTR NDA for oxycodegol, a NME full mu-opioid receptor agonist, for the management of chronic low back pain in adult patients with pain severe enough to require daily, around-the-clock, long-term opioid treatment.

The same committees will discuss Jan. 15, IntelliPharmaCeutics International Inc.’s IPCIF NDA for an extended-release oral tablet formulation of oxycodone. The investigational candidate is being evaluated for the management of moderate-to-severe pain when a continuous, around-the-clock opioid analgesic is needed for an extended period of time

The Anesthetic and Analgesic Drug Products Advisory Committee will review Jan. 16, DURECT Corporation’s DRRX NDA for bupivacaine extended-release solution for instillation, for the proposed indication of post-surgical analgesia.

https://www.benzinga.com/general/biotech/20/01/15053527/attention-biotech-investors-mark-your-calendar-for-these-january-pdufa-dates

Intercept Pharma cut to Neutral from Buy by Citi

Target to $140 from $85

https://www.benzinga.com/stock/ICPT/ratings

DHS to review state laws giving driver’s licenses to undocumented immigrants

Acting Homeland Security Secretary Chad Wolf on Tuesday called for a departmentwide study of how recent state laws allowing undocumented immigrants to obtain driver’s licenses impact federal enforcement capabilities, according to The Associated Press.

Wolf’s memo, which went to agencies including U.S. Customs and Border Protection and the Transportation Security Administration, follows a New York law authorizing licenses for undocumented immigrants and restricting data sharing with federal agencies. Similar laws exist in a dozen other states, including New Jersey, which passed a similar measure in December.

Wolf wrote in his memo that the department needed to be “prepared to deal with and counter these impacts as we protect the homeland.” He also asked each recipient to take account of what Department of Motor Vehicles information is already available and of any security consequences their operations could risk without that data, according to the AP.

“The Trump administration takes the mission of protecting the Homeland very seriously,” DHS spokeswoman Heather Swift told the AP, adding that these types of laws “make it easier for terrorists and criminals to obtain fraudulent documents.”

Approximately 265,000 immigrants in the Empire State, the majority in New York City, are projected to seek licenses under the new law over the next three years, according to the AP, citing research from the Fiscal Policy Institute.

The law still requires applicants to obtain permits and pass road tests. Authorities in states with such laws argue that they will improve traffic safety by reducing the number of uninsured people on the road.

https://thehill.com/homenews/state-watch/476466-trump-administration-takes-aim-at-laws-allowing-undocumented-immigrants

Expected quality benefit from hospital M&A not there – study

A new study published in the New England Journal of Medicine undermines the often-given benefit of hospital mergers and acquisitions – improvement in quality of services after the transaction.

The authors looked for evidence of quality gains based on four metrics collected by the Centers for Medicare and Medicaid Services (patient satisfaction, deaths within a month of admission, return trips to the hospital within a month of leaving and how often certain heart, pneumonia and surgery patients received recommended care) at 250 hospitals acquired in deals between 2009 and 2013.

The results showed that the quality of care either stayed the same or got worse after the deal, contradicting American Hospital Association-sponsored research conducted by Charles River Associates that found improved quality and lower revenue per admission in the first year post-tie-up.

In 2018, there were 90 deals in the hospital sector, down 23% from 2017’s 117 but up 80% from 50 transactions in 2009.

Other studies found higher prices after mergers. For example, a study published in 2018 in the Quarterly Journal of Economics revealed that prices rose 6% after nearby hospitals were acquired.

Hospitals, like drugmakers, are under fire for high prices. The Trump administration has proposed a rule requiring them to disclose negotiated rates with payers, a move that prompted a lawsuit from operators filed about a month ago.

Selected tickers: XLV, HUM, UHS, THC, HCA, CYH, UNH, WCG, MOH, CI, CNC, ANTM, CVS

https://seekingalpha.com/news/3529028-expected-quality-benefit-from-hospital-m-not-study