Biotech Investors: Mark Your Calendar For June PDUFA Dates

Despite the FDA tied up with COVID-19-related activities, May turned out to be a positive month for biopharma companies from the perspective of drug approvals. Three new molecular entities were approved during the month and several other drugs also received the nod.
Meanwhile, Blueprint Medicines Corp BPMC 1.68% faced disappointment at the FDA altar, as its NDA for avapritinib for treating fourth-line gastrointestinal stromal tumor was clamped with a complete response letter.
Here are the key PDUFA dates scheduled for the unfolding month.
Merck’s Antibiotic Combo Up Before FDA For Label Expansion

• Company: Merck & Co., Inc. MRK 1.41%
• Type of Application: sNDA
• Candidate: Recarbrio
• Indication: hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia
• Date: June 4

Recarbrio is a combination of imipenem, a penem antibacterial, cilastatin, a renal dehydropeptidase inhibitor, and relebactam, a betalactamase inhibitor, indicated for the treatment of complicated urinary tract infections and complicated intra-abdominal infections.

Viela Eyes Commercial Status With its Autoimmune Disorder Drug Approval

• Company: Viela Bio Inc VIE 2.71%
• Type of Application: BLA
• Candidate: inebilizumab
• Indication: neuromyelitis optica spectrum disorder, or NMOSD
• Date: June 11

The FDA accepted the BLA for the investigational anti-CD19 monoclonal antibody on April 27, 2019. NMOSD is a rare autoimmune disease characterized by unpredictable attacks that often lead to severe, irreparable disability including blindness and paralysis.
The company said in its first-quarter earnings release it has begun preparations for potential regulatory approval, hiring and training market access and sales teams and deploying MSLs. The company expects to commercialize the drug shortly after.

Can Merck’s Wonder Cancer Drug Snag Another Approval

• Company: Merck
• Type of Application: sBLA
• Candidate: Keytruda
• Indication: solid tumors
• Date: June 16
• Merck announced April 7 FDA acceptance of the application with priority review. Keytruda as a monotherapy is being evaluated for treating adult and pediatric patients with unresectable or metastatic solid tumors with tissue tumor mutational burden-high, as determined by an FDA-approved test, who have progressed following prior treatment and who have no satisfactory alternative treatment options

Ultragenyx Seeks Label Expansion For Partnered Drug To Treat Low Serum Phosphate Levels

• Company: Ultragenyx Pharmaceutical Inc RARE 1.67% and Kyowa Kirin
• Type of Application: sBLA
• Candidate: burosumab
• Indication: hypophosphatemia
• Date: June 18

The FDA accepted the application for priority review Feb. 27. Burosumab is a fully human monoclonal IgG1 antibody, which works against the phosphaturic hormone FGF23. This hormone reduces serum levels of phosphorus and active vitamin D by regulating phosphate excretion and active vitamin D production by the kidney.
It has already been approved for the treatment of X-linked hypophosphatemia in adult and pediatric patients 6 months of age and older. The companies are now seeking label expansion to include the indication FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized.

Epizyme Looks To Strike It Rich With Another Tazemetostat Approval

• Company: Epizyme Inc EPZM 2.45%
• Type of Application: sNDA
• Candidate: tazemetostat
• Indication: follicular lymphoma
• Date: June 18

Tazemetostat was initially approved for epithelioid sarcoma in January, and has been sold under the brand name Tazverik. The regulatory application, which was accepted for priority review, seeks approval for the drug for patients with relapsed or refractory follicular lymphoma who have received at least two prior lines of systemic therapy.

Can Second Time Be Charm For Nabriva?

• Company: Nabriva Therapeutics PLC – ADR NBRV 31.49%
• Type of Application: NDA
• Candidate: Contepo
• Indication: complicated urinary tract infection or cUTI
• Date: June 19

Nabriva’s original NDA was rejected by the FDA in April 2019, with the regulatory agency handing down a complete response letter on the pretext of issues related to facility inspections and manufacturing deficiencies at one of Nabriva’s contract manufacturers.
The company resubmitted the application in late December, and the FDA acknowledged the resubmission in mid-January.

Evoke Knocks The FDA Altar After A Prior Rejection

• Company: Evoke Pharma Inc EVOK 5.26%
• Type of Application: NDA
• Candidate: Gimoti
• Indication: diabetic gastroparesis
• Date: June 19

Evoke faced a rejection at the FDA altar once, and resubmitted the application, which was accepted for review in January. GimotI is a nasal spray product candidate for the relief of symptoms in adult women with acute and recurrent diabetic gastroparesis.

Karyopharm Blood Cancer Drug On Track For Second Approval?

• Company: Karyopharm Therapeutics Inc KPTI 7.41%
• Type of Application: sNDA
• Candidate: Selinexor
• Indication: relapsed or refractory diffuse large B-cell lymphoma
• Date: June 23

Karyopharm announced on Feb. 19 FDA acceptance of the regulatory application, which sought accelerated approval for oral Selinexor tablets for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma, not otherwise specified, who have received at least two prior therapies.
Selinexor was approved in July 2019 as a combo treatment option along with dexamethasone for the treatment of adult patients with relapsed refractory multiple myeloma, who have received at least four prior therapies.

Zogenix’s Hopes For No Jitters On Seizure Drug Review

• Company: Zogenix, Inc. ZGNX 1.48%
• Type of Application: NDA
• Candidate: Fintepla
• Indication: seizures associated with Dravet syndrome
• Date: June 25
• Zogenix’s regulatory filing for Fintelpa was accepted for priority review in November 2019, with a PDUFA date of March 25. The FDA extended the review period by three months to give itself time to look at the additional data provided by the company.

Heron Expects Gain From Pain Drug Review

• Company: Heron Therapeutics Inc HRTX 2.74%
• Type of Application: NDA
• Candidate: HTX-011
• Indication: post-operative pain
• Date: June 26

Heron’s HTX-011 is a combo drug consisting of bupivacaine and a low dose of non-steroidal anti-inflammatory drug meloxicam, and is a non-opioid pain drug. The NDA was originally submitted in October 2018, and in response to the application, the FDA issued a complete response letter in April 2019, citing the need for additional CMC and non-clinical information.
Heron resubmitted the NDA in October 2019, and in February the company said the FDA extended the review period by three months, rendering the PDUFA data on June 26.
If approved HTX-011 will compete with Pacira Biosciences Inc’s PCRX 1.43% Exparel, Guggenheim Securities analyst Dana Flanders said in a recent note. Citing the firm’s post-operative pain survey, Flanders said HTX-011 is likely to see significant growth at the expense of Exparel. The analyst said price point may be key in driving significant uptake.

Can Chiasma Cross The FDA Hurdle This Time Around?

• Company: Chiasma Inc CHMA 0.16%
• Type of Application: NDA
• Candidate: Mycapssa
• Indication: acromegaly
• Date: June 26

The FDA accepted Chiasma’s originally submitted NDA in August 2015.
Mycapssa, or octreotide capsules, is an oral drug being evaluated for the maintenance therapy of adult patients with acromegaly. A complete response letter was issued by the FDA in April 2016, seeking an additional clinical trial to establish the efficacy. Following a resubmission in Dec. 2019, the FDA accepted the application in January, giving it a PDUFA date of June. 26.
Acromegaly is a hormonal disorder that is caused by the production of too much hormone by the pituitary gland during adulthood, causing bone size to increase.

Intercept’s Wait For NASH Drug May Not End

• Company: Intercept Pharmaceuticals Inc ICPT 1.77%
• Type of Application: NDA
• Candidate: Obeticholic acid
• Indication: fibrosis due to non-alcoholic steatohepatitis
• Date: June 26
• NASH has become a tough nut to crack for biotech companies, with no approved drug yet despite a plethora of ongoing research. Intercept filed the NDA in November 2019 following positive Phase 3 results from the GENERATE study. The PDUFA date, which was originally fixed as March 26, was extended by three months.

However, dimming the prospects, the company informed earlier this month an Adcom meeting scheduled tentatively for June 9 was postponed by the FDA. The company, therefore, indicated the review period is likely to be extended.
https://www.benzinga.com/general/biotech/20/06/16135090/attention-biotech-investors-mark-your-calendar-for-june-pdufa-dates