Target $135
Thursday, January 2, 2020
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.
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.
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.
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.
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