Attention Biotech Investors: Mark Your Calendar For August PDUFA Dates

July turned out to be a mixed month for FDA approvals. Endo International PLC's ENDP cellulite treatment, Osmotica Pharmaceuticals PLC's OSMT eye disorder drug and Jazz Pharmaceuticals PLC's JAZZ drug to treat excessive daytime sleepiness were among those which successfully cleared the FDA hurdle.

A few others, including Verrica Pharmaceuticals Inc VRCA and Eagle Pharmaceuticals Inc EGRX, are forced to wait. The regulatory agency handed down a complete response letter for Verrica's viral skin infection treatment, while Eagle Pharma's PDUFA date for its exertional heat stroke drug was postponed until Aug. 8 due to FDA's reallocation of resources due to COVID-19.

Four new molecular entities, or NMEs, were approved in July, taking the total number of NME approvals for the year to 29.

Here are the key PDUFA dates scheduled for August.

GSK Seeks To Expand Use Of Triple Combo Drug To Asthma

• Company: GlaxoSmithKline plc GSK
• Type of Application: sNDA
• Candidate: Trelegy Elypta
• Indication: asthma
• Date: Aug. 2 (estimated based on the 10-review period from the regulatory application filing date of Oct. 2)

Trelegy Elypta is a single inhaled triple therapy consisting of fluticasone furoate/umeclidinium/vilanterol) and has already been approved for chronic obstructive pulmonary disease. The company is now seeking label expansion to include asthma as an indication.

The investigational asset is jointly developed by GlaxoSmithKline and Innoviva Inc INVA. Theravance Biopharma Inc TBPH has an economic interest in potential future payments from GlaxoSmithKline.

Adcom Cancellation Reduces Likelihood Of DBV's Peanut Allergy Patch Approval

• Company: DBV TECHNOLOGIE/S ADR DBVT
• Type of Application: BLA
• Candidate: Viaskin Peanut
• Indication: Peanut allergy in children, ages 4-11 years
• Date: Aug. 5

Viaskin Peanut is DBV's lead product candidate designed to potentially reduce the risk of life-threatening allergic reactions due to accidental exposure to peanuts. A non-invasive, once-daily, epicutaneous patch, Viaskin Peanut seeks to deliver microgram quantities of peanut antigen to activate the immune system.

The investigational immunotherapy, which has Breakthrough and Fast Track designations, was accepted for review in October 2019. The FDA communicated its intention to hold an Adcom meeting on May 15. With the agency canceling the meeting due to questions over patch's efficacy, including the impact of patch-site adhesion, the probability of approval has reduced.

Can Second Time Prove Charm For Trevena?

• Company: Trevena Inc TRVN
• Type of Application: NDA
• Candidate: Oleceridine
• Indication: moderate-to-severe acute pain
• Date: Aug. 7

Oliceridine is a G protein-selective mu-opioid receptor agonist in development for the management of moderate-to-severe acute pain in hospitals or other controlled clinical settings where intravenous therapy is warranted.

Trevena's original NDA was handed down a complete response letter in November 2018, with the FDA requesting additional clinical data on QT prolongation and indicating the submitted safety database is not of adequate size for the proposed dosing.

The company resubmitted the regulatory application in February, and the FDA deemed it as a complete response and assigned a PDUFA date of Aug. 7.

"Positive physician feedback builds a strong use case of IV oliceridine," HC Wainwright analyst Douglas Tsao said in a March note.

Eagle Pharma Awaits Decision On Heat Stroke Drug

• Company: Eagle Pharma
• Type of Application: NDA
• Candidate: Ryanodex
• Indication: exertional heat stroke, or EHS.
• Date: Aug. 8

Ryanodex, or dantrolene sodium for injectable suspension, is indicated for the treatment of malignant hyperthermia in conjunction with appropriate supportive measures. The original NDA for treating EHS was handed down a CRL in July 2017. Eagle resubmitted the application in Jan. 2020.

EHS, a severe form of heat-related illness, is characterized by core body temperature of 104° F or greater and significant neurological dysfunction, such as sudden changes in behavior, seizures or coma.

"We believe expectations for approval are low; thus, if EGRX is successful in gaining approval on or by the PDUFA date, we would expect this to be positively received given that it would provide upside not fully reflected in current estimates," Cantor Fitzgerald said in a recent note.

Second Try For Eton's Eye Inflammation Drug

• Company: Bausch Health Companies Inc BHC and Eton Pharmaceuticals Inc ETON
• Type of Application: NDA
• Candidate: EM-100
• Indication: allergic conjunctivitis.
• Date: Aug. 10

EM-100 is Eton's preservative-free ophthalmic solution for the treatment of allergic conjunctivitis. The company has executed a commercialization deal with Bausch Health to market the ophthalmic solution. Bausch Health, which had filed a regulatory application for EM-100, received a complete response letter for the application in July 2019.

Following a resubmission, the FDA assigned a PDUFA date of Aug. 10.

Fennec Waits to Hear On Drug To Treat Chemotherapy-induced Hearing Loss

• Company: Fennec Pharmaceuticals Inc FENC
• Type of Application: NDA
• Candidate: Pedmark
• Indication: chemotherapy-induced ototoxicity
• Date: Aug. 10

Pedmark is a unique formulation of sodium thiosulfate, and is being evaluated for the prevention of ototoxicity induced by cisplatin chemotherapy in patients one month to less than 18 years of age with localized, non-metastatic, solid tumors.

The company announced in April FDA accepted the regulatory application with priority review.

If approved, Pedmark could be the first product for the prevention of Cisplatin-induced hearing loss in children, Fennec said.

Roche Seeks Label Expansion For Asthma Biologic

• Company: Roche Holdings AG RHHBY
• Type of Application: sBLA
• Candidate: Xolair
• Indication: Nasal polyps

Date: Q3 (estimated to be Aug. 11 based on 10 months from the filing of regulatory application)

Xolair, an antibody, has been approved in the U.S. for the treatment of moderate-to-severe persistent asthma in patients 6 years and older and chronic idiopathic urticaria in patients 12 years and older.

Roche's Genetech unit is seeking to get approval for the additional indication of treating nasal polyps in adult patients 18 years of age and older with inadequate response to intranasal corticosteroids. If approved, Xolair would become the first antibody to help reduce the size of nasal polyps and help improve symptoms through targeting and blocking immunoglobulin E, the company said.

Gilead Awaits Nod For Rheumatoid Arthritis Drug

• Company: Gilead Sciences, Inc. GILD and GALAPAGOS NV/S ADR GLPG
• Type of Application: NDA
• Candidate: filgotinib
• Indication: Rheumatoid arthritis
• Date: Aug. 19 (estimated assuming a priority review)

Filgotinib, an oral, selective JAK1 inhibitor for the treatment of adults who are living with moderate-to-severe rheumatoid arthritis, is widely believed by analysts to possess blockbuster potential. Gilead had licensed the investigational asset from Galapagos.

Gilead submitted the NDA in December, along with a Priority Review voucher.

Go Or No-Go For BioMarin's Hemophilia Gene Therapy?

• Company: BioMarin Pharmaceutical Inc. BMRN
• Type of Application: BLA
• Candidate: Valoctocogene roxaparvove
• Indication: hemophilia A
• Date: Aug. 21

Valoctocogene roxaparvovec is BioMarin's investigational AAV5 gene therapy for treating adults with hemophilia A. If approved, it will become the first gene therapy for any type of hemophilia.

Tricida Staring Down the Barrel Ahead Of Vevermier PDUFA Date

• Company: Tricida Inc TCDA
• Type of Application: NDA
• Candidate: TRC 101
• Indication: metabolic acidosis in patients with chronic kidney disease
• Date: Aug. 22

Veverimer, codenamed TRC101, is a non-absorbed, orally-administered polymer designed to treat metabolic acidosis in patients with chronic kidney disease.

Casting doubts on decision coming by the scheduled date, the company said on July 15 it received a notification from the FDA, stating that the agency has identified deficiencies that preclude discussion of labeling and post-marketing requirements/commitments at this time.

There is little time left from now until the PDUFA date for the FDA to inform Tricida of the nature of the deficiency and for Tricida to subsequently respond, Needham analyst Alan Carr said, commenting on the FDA communication. A Complete Response Letter appears increasingly probable, he said.

However, if the company is requested to submit additional information prior to the PDUFA date, a major amendment, triggering a three-month extension of the PDUFA date may also be a possibility.

Roche Eyes SMA Drug Approval After 3-Month Delay

• Company: Roche and PTC Therapeutics, Inc. PTCT
• Type of Application: NDA
• Candidate: Risdiplam
• Indication: spinal muscular atrophy
• Date: Aug. 24 (expected)

Roche is leading the clinical development of risdiplam, as part of a collaboration with the SMA Foundation and PTC. Risdiplam is an investigational, orally-administered survival motor neuron-2 splicing modifier for spinal muscular atrophy.

Roche's NDA was accepted for Priority Review in Decemeber, with a PDUFA date of May 24. The FDA extended the review period by three months, reasoning that it needs time to review additional data submitted by Roche in February based on its discussion with the FDA.

Will Lipocine Succeed With Its Fourth Try?

• Company: Lipocine Inc LPCN
• Type of Application: NDA
• Candidate: Tlando
• Indication: Hypogonadism
• Date: Aug. 28

Tlando, a novel oral prodrug of testosterone containing testosterone undecanoate, is designed to help restore normal testosterone levels in hypogonadal men.

The investigational asset stumbled at the FDA altar three times in the past. Following Lipocine's third resubmission, the FDA has established a PDUFA action date of Aug. 28

Roche Hopes To Be Better Late Than Never

• Company: Roche
• Type of Application: BLA
• Candidate: Satralizumab
• Indication: neuromyelitis optica spectrum disorder, or NMOSD
• Date: Aug. 29

Satralizumab is an investigational humanized monoclonal antibody that targets the IL-6 receptor. The cytokine IL-6 is thought to be a key driver of NMOSD, triggering the inflammation cascade and leading to damage and disability.

Roche announced FDA acceptance of the BLA Oct. 30, and assuming a standard ten-month review period, the decision is expected Aug. 29.

Earlier this year, the Japanese drug regulatory agency approved satralizumab for treating NMOSD.

If the approval comes through, Roche may have to face competition from two drugs already in the market, namely Alexion Pharmaceuticals, Inc.'s ALXN Soliris and Viela Bio Inc's VIE Uplizna.

Morphosys-Incyte Await FDA Word On Lymphoma Drug

• Company: Incyte CorporationINCY and Morphosys Ag MOR
• Type of Application: BLA
• Candidate: Tafasitamab
• Indication: diffuse large B cell lymphoma, or DLBCL
• Date: Aug. 30

Tafasitamab is an investigational humanized Fc-engineered monoclonal antibody directed against CD19. Morphosys had licensed the investigational asset from Xencor Inc XNCR in 2010. In January, Morphosys and Incyte entered into a a collaboration and licensing agreement to commercialize tafasitamab globally.

The FDA accepted March 2, Morphosys' BLA, seeking approval for tafasitamab, in combination with Revlimid, as a treatment option for relapsed or refractory DLBCL. The agency set a PDUFA action date of Aug. 30.

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