Attention Biotech Investors, Here's Your PDUFA Primer For October

The biotech space can be an investor's delight, fetching staggering returns, while at the same time taking them through the throes of despair if bets go wrong — all courtesy of make-or-break events.

In September, the iShares NASDAQ Biotechnology Index (ETF) IBB has lost 0.44 percent and the NYSE ARCA BIOTECH INDEX is down 0.63 percent. In comparison, the tech-weighted NASDAQ Composite was down 0.90 percent, but the S&P 500 has rallied 1.01 percent. The performance of the biotech space has been somewhat in sync with the broader market, or at least technology stocks.

A Look Back At September PDUFA Events

September has been a month of plenty as far for PDUFA events are concerned.

The FDA has approved 33 NMEs, or new molecular entities, this year in contrast to 22 in the 2016 as a whole. 

In September alone, the FDA approved two NMEs: Solosec and Aliqopa. On Sept. 12, the FDA approved Symbiomix Therapeutics' Solosec to treat bacterial vaginosis in adult women.

Bayer AG (ADR) BAYRY's Aliqopa, a NME, gained FDA approval Sept. 14 to treat adults with relapsed follicular lymphoma.

Mylan N.V. MYL faced a setback in September, when the FDA extended the target data for its trastuzumab biosimilar until Dec. 3, 2017. The company and its Indian partner Biocon said the decision will not affect the timeline for commercialization of this product in the U.S.

On Sept. 14, Allergan plc Ordinary Shares AGN and Amgen, Inc. AMGN said the FDA approved their ABP 215 for multiple cancer indications' the drug is a biosimilar candidate to bevacizumab, sold under the brand name Avastin.

Neos Therapeutics Inc NEOS's ADHD treatment Adzenys ER, an extended-release amphetamine oral suspension, was approved by the FDA Sept. 15.

GlaxoSmithKline plc (ADR) GSK and Innoviva Inc INVA announced FDA approval Sept. 18 for their once-daily, single inhaler therapy fluticasone furoate/umeclidinium/vilanterol for the treatment of chronic obstructive pulmonary diease under the brand name Trelegy Ellipta for the treatment of chronic obstructive pulmonary disease. 

On Sept. 25, the FDA announced approval of Bristol-Myers Squibb Co BMY's Opdivo for treating hepatocellular carcinoma after prior sorafenib therapy.

Johnson & Johnson JNJ's Janssen Pharma unit failed to secure FDA nod for Sirukumab to treat rheumatoid arthritis. The decision announced Sept. 22 was in line with an August FDA Advisory Committee recommendation to reject the drug. Source: Y Charts 

Ushering In October's Events

1. Flexion's Lead Candidate Walks To The FDA Altar

Company name(s): Flexion Therapeutics Inc FLXN

Type of Application: NDA.

Drug/Treatment Candidate: Zilretta - FX006.

Indication: osteoarthritis of the knee.

Date: Oct. 6.

The company, specializing in developing treatment options for musculoskeletal conditions, is knocking the FDA doors for Zilretta, which employs proprietary microsphere technology combining triamcinolone acetonid, a commonly administered, short-acting corticosteroid, with a poly lactic-co-glycolic acid matrix. The NDA was accepted by the FDA in February this year.

2. Mylan Seeks October Rebound With Neulasta Biosimilar

Company name(s): Mylan.

Type of Application: BLA.

Drug/Treatment Candidate: MYL-1401H.

Indication: Reduce the duration of neutropenia (low count of neutrophils) and the incidence of fever associated with neutropenia in adult patients treated with chemotherapy in certain types of cancer.

Date: Oct. 9.

Mylan is locked in a legal battle with Amgen, the maker of Neulasta, or pegfilgrastim, with the latter claiming that the former has infringed two of the patents associated with its anti-infection treatment.

3. AcelRx Bracing For Pain Or Gain?

Company name(s): AcelRx Pharmaceuticals Inc ACRX

Type of Application: NDA

Drug/Treatment Candidate: ARX-04 (SAP303), branded as Dsuvia.

Indication: Treat moderate-to-severe pain in a medically supervised setting.

Date: Oct. 12.

ARX-04 is a 30 mcg tablets of sufentanil, a highly potent synthetic opioid delivered as often as once per hour under the tongue through a disposable, pre-filled, single dose applicator. The company announced in June the FDA notification that an advisory committee meeting is not planned for the NDA.

4. Neurocrine Seeks INGREZZA Approval For Additional Dosing

Company name(s): Neurocrine Biosciences, Inc. NBIX.

Type of Application: sNDA.

Drug/Treatment Candidate: INGREZZA 80 mg capsules.

Indication: Treat adults with tardive dyskinesia.

Date: Oct. 14.

The company received approval for INGREZZA in April this year for an initial dosing of 40mg once daily, which could then be increased after one week to 80mg once daily. The company is now seeking approval for another dosing. Tardive dyskinesia is a condition in which one cannot control body movements. 

5. Johnson & Johnson Banks On STELARA sBLA Approval To Ward Off Competition

Company name(s): Johnson & Johnson JNJ's Janssen Biotech unit.

Type of Application: sBLA.

Drug/Treatment Candidate: STELARA, or ustekinumab.

Indication: Treat adolescents (12 to 17 years of age) with moderate to severe plaque psoriasis.

Date: Oct. 15.

STELARA is a human monoclonal antibody that targets interleukin (IL)-12 and IL-23 cytokines. This has already been approved for treating adults with moderate to severe plaque psoriasis since September 2009. An approval is important for the company to keep the teeming competition at bay. It generated sales of $2.47 billion in 2015, according to Fierce Pharma.

6. Can Antares' Makena sNDA Deliver?

Company name(s): Antares Pharma Inc ATRS and AMAG Pharmaceuticals, Inc. AMAG.

Type of Application: sNDA.

Drug/Treatment Candidate: Makena Auto Injector.

Indication: Lower the risk of premature births.

Date: Oct. 17.

Makena is a hydroxy progesterone caporate injection given six days a week between 16 and 20 weeks of pregnancy to prevent premature birth.

7. Antares Seeks A Shot In The Arm With QuickShot

Company name(s): Antares Pharma.

Type of Application: NDA.

Drug/Treatment Candidate: VIBEX QuickShot.

Indication: Treat adult men with low testosterone associated with a diagnosed condition known as hypogonadism.

Date: Oct. 20.

VIBEX QuickShot is a drug-device combination product for the delivery of testosterone enanthate using a subcutaneous auto injector. Hypogonadism is a condition in which the body does not produce enough testosterone, the hormone that plays a key role in masculine growth and development during puberty, and maintenance of musculoskeletal, metabolic and mental health in maturity.

8. Johnson & Johnson's Awaits Twin Approval For RA Drug

Company name(s): Johnson & Johnson's Janssen Biotech.

Type of Application: sBLA.

Drug/Treatment Candidate: Simponi Aria, or Golimumab.

Indication: Treat adults with active psoriatic arthritis and adults with active ankylosing spondylitis.

Date: Oct. 21.

Simponi Aria is a fully human tumor necrosis factor (TNF)-alpha inhibitor and has already approved for use as an intravenous infusion to treat adults with moderately to severely active RA in combination with methotrexate.

8. Alexion Awaits Soliris Sunshine

Company name(s): Alexion Pharmaceuticals, Inc. ALXN.

Type of Application: sBLA.

Drug/Treatment Candidate: Soliris, or Eculizumab.

Indication: Treat refractory generalized Myasthenia Gravis patients who are anti-acetylcholine receptor antibody-positive.

Date: Oct. 23.

9. Will FDA Go With Panel Recommendation On Shingrix?

Company name(s): GlaxoSmithKline plc (ADR) GSK and Agenus Inc AGEN.

Type of Application: BLA.

Drug/Treatment Candidate: Shingrix vaccine, containing Agenus' adjuvant, QS-21 Stimulon.

Indication: Treat shingles in adults over 50.

Date: Oct. 24.

Earlier this month, a FDA committee voted unanimously that the data presented by the company support the efficacy and safety of Shingrix for the prevention of herpes zoster, or shingles, in adults 50 and older.

10. Can PTC Therapeutics Finally Succeed After Initial Hurdles?

Company name(s): PTC Therapeutics, Inc. PTCT.

Type of Application: NDA.

Drug/Treatment Candidate: Translarna.

Indication: Duchenne muscular dystrophy.

Date: Oct. 24.

Following the initial filing, the FDA issued a refusal to file letter in February 2016. An appeal was again rejected in October 2016. The decision to resubmit was made in the first quarter of 2017, with the FDA Advisory Committee's verdict due later this month.

11. Can Second Time Prove The Charm For TESARO's Rolapitant?

Company name(s): TESARO Inc TSRO.

Type of Application: NDA.

Drug/Treatment Candidate: Rolapitant IV.

Indication: For preventing delayed nausea and vomiting associated with initial and repeat courses of emetogenic cancer chemotherapy.

Date: Oct. 25.

After receiving a complete response letter in January, TESARO has made a resubmission for Rolapitant, an injectable version of its oral treatment Varubi.

Related Links:

What Do Versartis' Troubles Mean For Opko Health?

The Street Has Finally Recognized The Exelixis Clinical Program Potential